Ionis Pharmaceuticals is currently seeking trial participants to evaluate an experimental DM1 drug, IONIS-DMPKrx, that may help modify the disease rather than just treat the symptoms.
Rare Disease Day on Feb 29th gives us an opportunity to shine a spotlight on DM. MDF has activities planned for the entire month of February. Find out how you can join us in speaking out about DM.
The use of anesthesia raises special risks to DM patients, which include heightened sensitivity to sedatives and analgesics. Serious complications are most common in the post-anesthesia period when risk of aspiration and other complications are increased.
MDF has published two versions of its 2016 Anesthesia Guidelines:
- Click here for a handy, one-page summary of the anesthesia guidelines to share with your clinician and anesthesiologist.
- Click here for the complete "Practical Suggestions for the Anesthetic Management of a Myotonic Dystrophy Patient".
New to DM? For here for more information.
Word came from the FDA on January 14th that it would not approve the new drug application for "Kendrisa," Biomarin's antisense-oligo based drug for Duchenne muscular dystrophy.
End of year donations were more generous than ever this year, inspired in part by a $15,000 match contributed by the Boekelmann family, bringing us to over $1.1M in total 2015 contributions. A huge thank you to each and every one of you for being part of this special community and helping to sustain it. Happy New Year!