When Chris and Patrick were diagnosed, there was no genetic testing. No therapies were targeted to the “CODE” and no way to DECODE Duchenne. We made assumptions about carrier status and lived many years with only assumptions. I thought my mother was a carrier. I knew I was a carrier. The doctor made that assumption for me. Serial CKs were done on our daughters with the…

We are excited to announce that Parent Project Muscular Dystrophy and Sarepta Therapeutics are joining forces on a genetic testing program for patients with Duchenne or Becker muscular dystrophy. Through the new genetic testing program, called Decode…

We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for rare disease versus treatments for treat common…

I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might improve the clinical trial process. Highlighting the…

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.

Next Generation Exon-Skipping

Several laboratories presented data on ways…

Flu season is back and the vaccine is slightly different this year:

The “nasal spray flu vaccine” is a live attenuated vaccine, which contains living, but weakened virus.  It is generally not recommended for people with neuromuscular diseases on or off corticosteroids.  It is safe for caretakers, siblings and classmates who are…