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MDF at 2017 BIO International Convention

MDF staff recently attended the 2017 BIO International Convention hosted by the Biotechnology Innovation Organization (BIO). BIO offers a partnering track to patient advocacy groups, enabling meetings with companies having potentially mutual interests.

Preclinical Data Behind the Ionis Trial Published

Preclinical animal efficacy data used to support the scientific rational for Ionis’ phase 1/2 clinical trial in DM1 was just published.

New Drosophila Models for DM1 and DM2

New fly models show a DM2 phenotype at least as severe as DM1 and may provide a platform for studies of genetic modifiers and candidate therapy screening in DM.

DM1 Genotype and Cardiac Phenotype

A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.

Do We Treat DM as a Brain Disease?

A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.

NIH Update: Grant Advice

MDF strives to help National Institutes of Health (NIH) grant applicants compete for research funding.

The Path We’re On: Many Challenges, but Ultimately a Drug

Taking as many shots on goal as possible is the only path to approval and reimbursement of safe and effective drugs for DM.

DM Highlights at American Academy of Neurology Annual Meeting

MDF staff recently attended the 2017 annual meeting of the American Academy of Neurology, in Boston, MA. Here are highlights from that meeting.

Inheritance of CDM

There have been new discoveries in the way that congenital myotonic dystrophy (CDM) is inherited.

Telling the Quacks from the Cures

MDF is also happy to help you understand whether something is in a legitimate clinical trial, an approved therapy…or not.

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