The Jensen family has a long tradition of hosting an authentic Louisiana crawfish boil with live music for their family and friends in San Diego. The Crawfish Boil became a fundraiser in 2012 when Taylor Jensen and her young son, River, were diagnosed with myotonic dystrophy type 1 (DM1). The Jensen's learned first-hand how complex DM is and how it impacts an entire family. After struggling with the initial impact of the diagnosis, Taylor and Eric evolved the family tradition into a fundraiser to support Care and a Cure.

The Fundraiser has two ultimate goals: to raise awarness of the most common form of muscular dystrophy known as myotonic dystophy (DM); and to to raise funds to continue the research that will enable treatments and an eventual cure. This year marks the first time people living with DM are receiving a potential therapy. The first DM clinical trial began this year, evaluating a medication that targets not just disease symptoms but the genetic mutation that causes the disease.

This year the Jensen Family had their 5th Annual Crawfish Boil Fundraiser ("Pinching Tails for a Cure") hosted at the San Diego Harley Davidson Dealership. Thanks to the sponsors and donors, the Crawfish Boil was a success, raising over $30,000 - bringing that to a total of $110,000 over the last five years!

You can read more about their personal journey with DM in this letter from the Jensen family.

Are you interested in hosting a fundraiser for Care and a Cure? MDF is here to help.

Many MDF community members are aware of an important, multi-year MDF effort -- MDF 3.0: Accelerating Drug Development -- because we published an article on the launch of this initiative in early 2015.

As you may know, the goals of MDF 3.0 are to:

• Strengthen capacity and infrastructure in DM clinical care to drive more accurate clinical trial design, improve capacity to evaluate drug efficacy and advance understanding of disease course
• Deepen and strengthen the research and development bench to drive more myotonic dystrophy discoveries
• Expand the drug development pipeline with additional pharamceutical partners, additional translational research and more critical data
• Incentivize investment in myotonic dystrophy via key studies, data collection, industry partnerships and a targeted and immediate advocacy effort with federal agencies and legislators

Below is a first-year report on how elements of MDF 3.0 got launched and how we are doing to date.

1.   Expand the Fellowship Program

First launched in 2009, MDF's Fund-a-Fellow (FAF) program was designed to:

• Attract new investigators to the field of myotonic dystrophy (DM) research
• Increase interest and activity in the field of myotonic dystrophy research
• Expand knowledge and understanding of DM
• Increase the number of labs and academic programs engaged in or expanding their engagement in DM research

To date, MDF's FAF Program has funded seventeen postdoctoral research students, providing over $1,700,000 to DM research focused on enhancing the quality of life of people living with DM and advancing research focused on finding treatments and a cure for this disease.  

And for 2016, an unprecedented six new fellows have been funded, who are pursuing research projects ranging from understanding the cognitive effects of DM to making a mouse model of DM2.

2.  Conduct a Prevalence Study

A major objective of MDF 3.0 is to determine how common the DM1 and DM2 mutations are in the general population. Many have quoted the figure “1 in 8000” as the “prevalence” of myotonic dystrophy—that is, how many people may have the disease at any given time—but this number comes from studies done in other countries, and it also relies on diagnosed cases of DM. Given that the diagnostic odyssey can take as long as 14 years in myotonic dystrophy, it is likely that diagnosed cases represent the tip of the iceberg.  

An accurate understanding of the occurrence of DM in the population, both diagnosed and undiagnosed, is a fundamental building block for efforts to improve treatment resources and develop a cure for DM. In 2015 MDF released a “Request for Applications” to fund the first of a two-part project to understand how common these mutations are in the population.

In October, MDF announced that funding for the first phase of this project will go to Dr. Nicholas Johnson at the University of Utah. Dr. Johnson will focus on making sure that he can scale up his diagnostic testing method to process 50,000 or more total samples at a reasonable cost.  

In the next phase of the project, which will be announced in the spring of this year, MDF will invite investigators to submit their plans for screening a random sample of the population for mutations that cause DM1 and DM2. Results are expected in early 2017.

3.  Create access to tissue samples 

Early in 2015, MDF began hearing from members of industry that they were having difficulty obtaining biological samples, particularly cell lines, from people with DM that they could use to identify drugs that might be developed into therapies.

To help address this problem, MDF partnered with Orig3n, a new biotechnology start-up company that is specializing in creating cell lines from rare disease patients. Under a memorandum of understanding (MOU) between MDF and Orig3n, the company came to the 2015 MDF Annual Conference and drew blood from approximately 50 volunteers. The company intends to create cell lines over the next 12 months. The majority of Orig3n samples are from current Myotonic Dystrophy Family Registry members and can be tracked to their registry records anonymously through a unique identifier number, providing useful additional information about the cell line donors.   

MDF is working with Dr. John Day’s group at Stanford University and other investigators with biobanks to raise visibility and promote more community and researcher participation in them.

4.  Drive drug discovery through an assay development and screening project 

In June MDF issued a Request for Applications to help identify possible new drugs for myotonic dystrophy. This led to collaboration between academic investigators with in-depth DM knowledge and industry and industry/government entities with drug development experience and scale-up capabilities.

A panel of experts in drug screening and therapy development reviewed the proposals submitted and chose the team that will receive a $200,000 award over the next year. The successful applicants will be announced in the next issue of the DM Research News.

5.  Accelerate identification of appropriate DM biomarkers and clinical endpoints

Biomarkers and clinical endpoints are different ways of measuring changes that occur as a consequence of a disease, over the course of a disease or in response to an intervention.  A biomarker is typically evidence of a physiological change that has occurred, often detected in blood or tissue samples, that is relevant to the disease process. A clinical endpoint is some way of measuring how a patient “feels, functions or survives.”  

Biomarkers that provide early evidence that a drug is working are crucial for making drug development economically feasible. This is especially true for diseases that progress relatively slowly, like DM.

Biomarkers are also useful in creating better trials (by selecting people with DM who have specific characteristics for trials or making sure those characteristics are balanced in the different groups that make up the trial).  

MDF held a meeting in Fall 2014 to explore the status of biomarkers and endpoints for myotonic dystrophy, addressed biomarkers at the FDA regulatory workshop in the Fall of 2015 and will issue a Request for Applications in Spring 2016 to provide support for the development or refinement of biomarkers for myotonic dystrophy.  

In addition to the need for biomarkers, the need to identify an appropriate primary clinical outcome measure for new therapeutics is becoming an urgent priority as the DMPKx phase I/II clinical trial moves toward completion.   

The primary clinical endpoint is the test that will be used by the Food and Drug Administration to determine if an experimental treatment actually works. For example, a clinical endpoint could be the time it takes someone to get up from a chair and walk 30 feet, or it could be a person’s score on a survey about the severity of his or her symptoms. The scientific community has proposed various endpoints.  

In November 2015 MDF released a request for applications to support projects to develop new endpoints for myotonic dystrophy or to further refine endpoints already in development. Successful applicants must demonstrate how they will work closely with FDA staff to ensure that the work will be useful from a regulatory standpoint. Successful applicants will be announced in late Spring 2015.

6. Federal Advocacy

Regulatory Advocacy

As part of the three year 3.0 effort, MDF launched a significant advocacy effort targeting US congressional leadership, federal agencies involved in the drug review and approval process, agencies leading research investment and policy, and international bodies with impact and influence on the drug development and approval processes. 

MDF hosted the first-ever meeting of stakeholders involved in drug development to look at the regulatory pathway and better understand how potential DM therapies will be reviewed and assessed by the Food and Drug Administration (FDA), the European Medicines Agency (EMA) and others. More than 80 participants from academia, industry, the FDA and other federal agencies joined MDF in September 2015 to review progress toward the development of biomarkers and endpoints for DM clinical trials, the design of clinical trials, and to hear feedback and information from the FDA to help guide their drug development efforts. 

As a part of that meeting, MDF also contracted with Silicon Valley Research Group to conduct the first-ever benefit-risk study for myotonic dystrophy potential therapies, to introduce first-phase information on what therapy benefits are most important to DM patients and what risks patients would be wiling to accept for those benefits. The findings from this study were presented at the FDA workshop at the suggestion of the FDA. 

Because the patient perspective is becoming increasingly important in the overall drug development and approval process, this benefit-risk study will be followed with other MDF efforts to present the patient voice and perspective to the FDA and other regulatory and federal bodies in the months ahead.

The meeting helped industry get current thinking from the FDA, and was followed by a half-day workshop of just industry and academic participants to discuss what they heard, explore common areas of potential partnership or barriers to progress. MDF is working with a professional science writer to finalize a draft publication of the proceedings from the all-day workshop, and plans to submit it for publication in early 2016. 

Legislative Advocacy

In early 2015 MDF hired a public policy firm in WA, DC to help define and deliver a comprehensive strategy to drive Care and a Cure through 2017. A key recent effort involved crafting report language and direction that will be proposed for insertion into the 2016 US federal budget to influence research investment and other policy work that is important to the DM community. MDF also engaged the team to bring congressional leadership to the first-ever legislative briefing MDF held on Capitol Hill in conjunction with our 2015 MDF Annual Conference and Hill Day, and to develop the materials used to educate Congress about critical DM issues. MDF will continue this work in 2016 with outreach and education efforts with key congressional leaders and additional grassroots and grasstops advocacy work in partnership with the MDF community.

Federal Agency Advocacy

People living with DM often struggle with the application process for Social Security disability benefits. MDF investigated and then met with the Social Security Administration (SSA) to explain the issues we experience and how the SSA can improve the process and outcomes for people with DM. MDF staff, DM physician Dr. Ami Mankodi and DM community members (Larry and Hunter Lord, and Loraine Dressler) participated in the meeting.

MDF will build on this work with education programs for the 2,500 SSA staffers who review claims in the field, and will create a Toolkit for community members to make the application process easier.

MDF also attended a number of Muscular Dystrophy Coordinating Committee (MDCC) meetings, applied for a position on the MDCC coordinating committee, supplied several rounds of proposed edits to the current MD CARE Act Action Plan (which the MDCC oversees), and held partnership meetings with key federal agencies including the National Institute of Neuromuscular Disorders and Stroke (NINDS) and other National Institutes of Health (NIH) agencies that influence the advance of DM science and research funding.

Grassroots and Grasstops Advocacy

MDF built on its first ever grassroots advocacy efforts in 2014 with a second Hill Day, bringing more than 80 community members to Capitol Hill to educate legislators and their staff on issues important to the DM community.  This work will expand significantly in 2016 with one-on-one meetings with key policy makers.

In short, 2015 was a great kick-off year for MDF 3.0, and we look forward to more high-impact work in 2016. We will keep you apprised.

Questions?

Please contact MDF. We would love to hear from you.

Generating "Benefit/Risk" Information for Drug Assessment

As part of our investment in the development of effective treatments for myotonic dystrophy, MDF is helping develop what is called "benefit/risk" information for regulatory agencies reviewing potential therapies. We need to better understand how people with DM weigh the benefits of new treatments against the risks these therapies might pose. To do this, MDF worked with Silicon Valley Research Group to conduct a survey that presented a series of hypothetical new treatments and asked that MDF community members choose the drug benefits that were most important, and what what they thought of potential side effects (some readers may recall having received an email solicitation for this survey over the summer). 

This type of analysis is called “Max-Diff Analysis” or sometimes “Best-Worst Scaling” and has been used in other benefit-risk studies and by the Food and Drug Administration (FDA) to develop "benefit/risk" information.  This method allows us to use robust statistics to determine, on average, what risks people living with myotonic dystrophy are or are not willing to accept for a given therapeutic benefit. The FDA and other international regulatory agencies are very interested in this information. 

A special thank you goes out to the 267 of MDF community members with DM1 and DM2 who met the entry criteria for the survey and soldiered through what may have felt like a lengthy and repetitive series of questions.  

What We Learned

The survey showed that reversing, stopping and slowing the progression of muscle weakness were the most preferred benefits, in that order. The side effects community members were most willing to tolerate overall for any therapy benefit were loss of appetite and a small increase in tiredness.  

People in the study also completed a short survey to rate the severity of their myotonic dystrophy.  Scores were grouped into mild, moderate and severe categories. For the majority of benefits, those with all levels of severity were similar in their willingness to tolerate side effects, except that those with more severe myotonic dystrophy were less willing to risk liver failure for any type of therapeutic benefit. Also, those with the highest severity rating for their myotonic dystrophy were more willing to tolerate an increase in tiredness if the drug could stop or reduce myotonia. The data reported here are based on the survey responses from those with DM1. The responses from those with DM2 are being analyzed now.

What's Next

These results were presented on September 17th at the MDF-sponsored all-day regulatory workshop on therapeutic development for myotonic dystrophy, which was attended by FDA staff, members of industry and the academic community. Next steps will likely include an in-depth follow-on study that looks at the benefit-risk preferences of caregivers and younger people with myotonic dystrophy. MDF is also investigating ways to collect “qualitative” data, such as stories and open-ended comments, regarding the benefit-risk preferences of those with myotonic dystrophy. Ultimately this information will be made available to FDA reviewers to help ensure that the opinions and preferences of those with myotonic dystrophy and their families are included the approval process for new therapies.

On September 17, 2015 MDF sponsored a regulatory workshop in Washington D.C. featuring speakers from academia, industry and the Food and Drug Administration (FDA).  As the therapeutic pipeline for myotonic dystrophy has started to fill, the “Myotonic Dystrophy Patient-Centered Therapy Development meeting” sought to explore the development of meaningful and measurable DM clinical endpoints and biomarkers, and to guide and advance the design of clinical trials for the care and cure of patients with myotonic dystrophy.  Click here for the agenda for that meeting, which was moderated by former FDA Deputy Director Dr. Stephen Spielberg, along with links to speaker presentations.

Introductions

Dr. Spielberg made a few introductory remarks at the meeting, noting that we are in an age of revolutionary treatments for rare, genetic diseases and that successful treatment development for these diseases is dependent on patient and family advocacy.

Dr. Spielberg introduced Dr. Richard Moscicki, Deputy Center Director for Science Operations in the Center for Drug Evaluation and Research (CDER) at FDA, who gave an overview of the FDA’s history with orphan drugs.  He noted that more orphan drugs were approved in 2014 than ever before and that the FDA is willing to be very flexible in its approach to serious rare diseases with unmet needs.

Living with Myotonic Dystrophy

Dr. Spielberg introduced a ten minute video produced by MDF featuring individuals with myotonic dystrophy who described what it is like to live day-to-day with the disease, dealing with gastrointestinal symptoms that sometimes left them homebound or unable to eat, myotonia of the tongue and facial muscles that made it difficult for them to communicate, and cognitive symptoms that left one young woman wishing that she “were cleverer.”

The video was followed by a short presentation by Dr. Sarah Clarke, a physician who self-diagnosed and then diagnosed both her young daughters, who are more severely affected.  Dr. Clarke described the helplessness of knowing that she was gradually becoming more affected herself, leaving her husband, also a physician, to be the caretaker for the entire family.  She wondered what would happen to their daughters when they were gone.

Dr. Nicholas Johnson, Assistant Professor of Neurology, Pathology and Pediatrics at the University of Utah then gave an overview of myotonic dystrophy pathology and symptoms, and also provided a look at some new data from his group on the frequency of comorbidities.

Session One: Real World Patient Data

The first of the main meeting sessions explored “Real World Patient Data”—what we have learned from studies and surveys about what people affected by myotonic dystrophy view as their most burdensome symptoms and what therapeutic benefits they would value most highly, as well as what risks they were willing to tolerate for new therapies.

Dr. Richard Moxley described key findings from the University of Rochester PRISM-1 study initiated by Dr. Chad Heatwole to better understand symptom burden.  The data from the PRISM-1 study showed that there was a marked increase in frequency and impact of mobility issues between ages 25 and 35, and that, while the most prevalent symptom was problems with hands and arms, the most impactful symptom was fatigue.

Dr. Katherine Hagerman of Stanford University presented data from the “Christopher Project”, a mailed survey about myotonic dystrophy with over 1400 respondents sponsored by the Marigold Foundation. The survey highlighted that muscle symptoms and fatigue were the heaviest symptom burden and provided insight into numerous aspects of daily living with the disease.

Dr. Sharon Hesterlee, MDF’s Chief Science Officer, presented data from a pilot study that takes a quantitative approach to measuring benefit/risk preferences of those with myotonic dystrophy. The study analyzed the responses of 267 people with adult-onset myotyonic dystrophy about hypothetical treatments with benefits related to relief from muscle weakness, fatigue and myotonia, and symptoms related to fatigue, GI symptoms, and liver damage. The results showed that people with myotonic dystrophy found hypothetical treatments that could reverse, stop or slow muscle weakness to be of greatest value, while treatments that did the same for fatigue to be of least value. Respondents rated liver failure to be the worst risk.

Ms. Pujita Vaidya from the Office of Strategic Programs at CDER, FDA described the Program’s efforts to conduct a series of Patient Focused Drug Development (PFDD) meetings to better understand patient needs and priorities. The results of these meetings are used to complete the first two rows of CDER’s Benefit-Risk Framework, which in turn is used in making regulatory decisions. Although myotonic dystrophy was not selected as one of the disease areas for CDER’s official PFDD program, Dr. Vaidya pointed out that CDER encourages the development of externally-led meetings that use the official PFDD meetings as a template.

Session Two: Myotonic Dystrophy Trial Endpoint Selection

Dr. Charles Thornton of the University of Rochester described what his group has learned from a three year study of functional measurements in 58 people with myotonic dystrophy. He showed that there was a large variance between all subjects with all measurements; that the 30 foot walk/run had the greatest reliability among measurements to show decline; that grip strength is also a sensitive measurement for progression, although it has a floor effect; and that composite myometry of mid-limb and distal muscles may have advantages for detecting improvement in moderately and heavily affected muscles.

Dr. Cynthia Gagnon of the Université de Sherbrooke described the Saguenay Longitudinal Study with over 15 years of follow-up data on 125 people with myotonic dystrophy. The goal of this study is to have a more comprehensive model to understand the relationships between impairments, disabilities, activities and participation in DM1. For example, Dr. Gagnon was able to show that lower extremity strength reliably correlates with the degree of mobility-related participation in social activities.

Dr. Nikunj Patel of the Clinical Outcomes Assessment Staff in CDER’s Office of New Drugs described the FDA’s approach to outcome measure development or selection. Dr. Patel explained that clinical outcome assessments must measure the right thing, in the right way, with the right amount of reliability to be able to say that there is a clear treatment benefit. He provided numerous links to appropriate FDA Guidances.

Session Three: Trial Design for a Slow-Progressing Heterogeneous Disease

Dr. Ronald Farkas from the Division of Neurology Products at CDER discussed clinical trial design for slowly progressing, heterogeneous rare diseases. In general, he explained, the FDA is very flexible about things like the size of the trial, the size of the safety database, and the length and number of trials needed to submit a New Drug Application (NDA) as long as the studies are conducted rigorously and the results are very clear-cut and convincing. He also made the point that there is not currently an FDA “preferred endpoint” for myotonic dystrophy and that many different types of measurements could be considered.

Session Four: Candidate DM Biomarkers

Dr. John Day of Stanford University provided an overview of biomarker development for myotonic dystrophy. He showed that candidate biomarkers for DM currently include myotonia, cardiac conduction changes, gastroparesis, endocrine/metabolic changes, CNS functional changes and transcriptome panels.

Dr. John Carulli of Biogen Idec discussed muscle and blood-based biomarkers for myotonic dystrophy clinical trials, pointing out that we are beginning to understand sources of splicing biomarker variability, which allows us to distinguish therapy-related changes from normal fluctuation. He feels that we still need to understand which markers are most sensitive to CUG repeat changes, that we need a statistical framework by which to interpret changes in splicing patterns and that we need to better correlate molecular markers with clinical measures of disease manifestation.

Dr. Shashi Amur of the Biomarker Qualification Program in CDER’s Office of Translational Sciences described the difference between qualifying a biomarker for use in multiple drug development programs and using the biomarker in a single drug development program. Qualifying a biomarker is a very structured process that typically takes between three and a half and five and a half years. Dr. Amur provided numerous references to guidance from the FDA on the biomarker qualification process and suggested that early engagement with the FDA on biomarkers is encouraged. She emphasized that qualification is not required to use a biomarker in a single drug development program.

Conclusions

Dr. Spielberg summarized the day’s meeting, noting particularly that there is a need to develop earlier predictors of efficacy and to define go/no-go decision making points to cut time and effort and limit risk. He also noted the need to better define biomarkers and expedite studies, pointing out that the FDA has regulatory but not disease area experience and is listening to the experts.

Meeting agenda and presentation links.

Many in the research, biomedical and patient advocacy communities have been tracking and advocating on behalf of the 21st Century Cures Act (H.R. 6). The legislation is intended to accelerate the discovery, development, and delivery of therapies via increased funding and revenue stability for research agencies, a more collaborative and efficient research environment for basic and translational science and a streamlined and accelerated regulatory review environment. The bill also includes provisions to improve patient access to quality healthcare and some initial attempts at pricing transparency.

In July, 21st Century Cures was passed by the US House of Representatives by a vote of 344 to 77 and forwarded to the US Senate for review. Many of the more hotly-contested sections of the bill, including those focused on longer protections from generic competition for both new and follow-on drugs, have been removed.

The Senate has drafted its own version of the bill, currently entitled, “Innovation for Healthier Americans.” From here the process of merging the two bills - and developing and voting on a final version - is likely to conclude in early 2016.

Understanding 21st Century Cures: The 3 Ds

Discovery

The 21st Century Cures Act increases funding for the National Institutes of Health by establishing the NIH Innovation Fund, a five-year project providing $1.75 billion per year to support early stage researchers and “higher risk, higher reward” research efforts. Additional provisions seek to enable easier, faster sharing of information among researchers, including electronic health records, and to streamline the clinical trial process by, for example, removing barriers to the use of central IRBs across multiple trial sites.

Development

The lengthiest section of the bill is devoted to improving the FDA regulatory process for drug development from “bench to bedside” by:

• codifying a framework at the FDA for the submission, review and qualification of biomarkers;
• allowing the sponsor of a drug to request that the FDA agree to an accelerated approval plan;
• authorizing the FDA to rely upon data previously submitted by a drug’s sponsor for a different drug that uses a similar underlying approach;
• encouraging better capturing of patient preferences, as in benefit/risk assessment; and
• enabling more efficient regulatory process surrounding the release of technology, including apps, for the better management of disease.

Delivery

To improve access to quality health care, 21st Century Cures promotes:

• interoperable systems to better enable the sharing of patient information throughout the health care system;
• electronic information and telecommunications technologies (telehealth programs) to support long-distance clinical health care and improve access to specialists. Insurance plans are increasingly expected to cover telehealth services; and
• some Medicare pricing transparency, beginning with what Medicare pays for products and services, access & efficiency. However, the larger issues of drug pricing transparency, how to manage exploding pharmaceutical costs, and who pays for the cost of innovation, have been largely avoided.

21st Century Cures and its sister bill in the Senate are expected to culminate with a final piece of legislation that has the potential to significantly enhance the drug discovery, development and approval process and improve patient access to quality care. The timing is good for myotonic dystrophy stakeholders. Our jobs at this point are to monitor the legislative process and continue to advocate for those outcomes that will enable DM disease stakeholders to improve the quality of life of DM patients and families. If you are attending the 2015 MDF Annual Conference in mid-September, consider signing up for our grassroots advocacy Hill Day, where we will organize over 70 family members, researchers and industry representatives into teams to visit congressional offices and advocate for 21st Century Cures and other DM-friendly legislation.

A section-by-section summary of the bill and related materials can be found on the House Energy & Commerce Committee website.

On Twitter, you can follow the dialog through the summer and fall by tuning into the hashtag #CURES2015.

08/18/2015

In 1982 the Food and Drug Administration (FDA) created the Office of Orphan Product Development to incentivize pharmaceutical companies to develop treatments for rare or 'orphan' diseases. The program has led to the successful development and marketing of more than 400 drugs and biologic products for rare diseases since 1983.

The Orphan Drug Designation Program provides orphan status to experimental drugs and biologics that are intended to safely and effectively treat, diagnose or prevent rare diseases. Rare or orphan diseases are defined as those that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 people but which are unlikely to enable pharmaceutical companies to recover the costs of developing and marketing drugs to treat them. 

Orphan Drug Designation qualifies the company sponsoring the drug for various development incentives, such as tax reductions and credits for qualified clinical testing, filing fee waivers, and the exclusive rights to the treatment for the specific rare disease for a period of seven years post-approval.

These incentives encourage companies to enter a market where the high costs of drug development are less likely to be recouped quickly due to the smaller pool of individuals needing the treatment and the expenses associated with developing and marketing treatments for rare diseases.

Designation as an Orphan Drug does not alter the standard FDA regulatory requirements for drug safety and efficacy that must be established through adequate and well-controlled clinical studies, nor does it mean that the drug is safe and effective or legal to manufacture and market in the United States. It means that the company developing the drug has qualified for certain incentives from the federal government.

For more information on clinical trials, visit the MDF Study & Trial Resource Center.

02/26/2015

On Friday, September 26, 2014, the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Amendments were signed into law by President Obama! Nearly 150 bipartisan Senators and Representatives cosponsored this bill, and it passed both chambers of Congress unanimously.

The MD-CARE Act has been extremely influential for the muscular dystrophy community. In the 14 years since the original bill's passage in 2000, federal funding for the muscular dystrophies has increased from $17 million to $75 million, with the pharmaceutical industry and private investments also contributing to the promising current state of science and research.

Over the past eight months, MDF community members have introduced Congress to myotonic dystrophy and educated legislators about the importance of passing this bill. You have written letters, visited district offices, made phone calls, shared your voice via SpeakingPhoto, and even held meetings with your members of Congress and their staff in Washington, DC. Additionally, the bill's sponsors, Senators Amy Klobuchar (D-MN) and Roger Wicker (R-MS), and Representatives Michael Burgess (R-TX) and Eliot Engel (D-NY), have worked tirelessly to ensure that the muscular dystrophy landscape is changed forever. Without the support of so many Americans living with muscular dystrophy, especially DM, this bill would not have had the successes that it has enjoyed.

Please take a moment to thank your Senators and Representative for passing the MD-CARE Amendments! Even if you have already written to them as part of our 500 Voices Campaign, continuing to follow up and establish relationships with them is critical to our future advocacy efforts.

MDF and its partners will continue to work with Congress and federal agencies to ensure that the recent amendments are properly implemented, and that our community continues to be addressed and prioritized in the future.

Thank you for all your hard work and dedication to making sure the MD-CARE Amendments passed. We are truly changing the face of myotonic dystrophy, and could not do it without your support!

09/26/2014