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!
MDF just kicked off the new year with our annual two-day strategic planning offsite. The planning team included DM researchers, board members and staff. Read on for some highlights of our plans for 2016 and progress to date on our 3-year drug development acceleration effort, MDF 3.0: Driving Drug Development.
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. Read a full report on the first-year results of MDF 3.0: Accelerating Drug Development, and the initiatives we are pursuing to promote the development of new DM therapies.
Established by the MD CARE Act in 2001, the Muscular Dystrophy Coordinating Committee (MDCC) is a federal advisory committee composed of representatives from major government agencies relevant to muscular dystrophy and a selection of “public” members representing patients and disease advocacy associations. The Commi