Note: The article in the Journal for Clinical Studies can be found here and begins on P. 60. This is a time of great potential for those living with neuromuscular diseases (NMDs). The pace of progress in science is accelerating and transformative therapies are on the horizon. In just the last few years, the FDA has . . .
To learn more about this report, please read our press release. Neuromuscular diseases encompass a broad group of disorders that are individually rare but collectively impact an estimated 250,000 patients in the United States, breaking the rare disease threshold of 200,000 individuals. Currently, treatment options for these diseases are limited. But the financial impact is staggering; . . .