muscular dystrophy

Elevated CPK: No short cut to muscular dystrophy diagnosisA pediatrician with special training in neuromuscular disorders cautions that nothing replaces the traditional workup—a complete history and physical—for diagnosing neuromuscular complaints.
Incremental clinical advances for Duchenne Muscular DystrophyJust last year, the FDA approved Exondys 51 (eteplirsen) for DMD, over the recommendations of FDA scientists and an external scientific advisory committee. Read more.
FDA Approves Drug to Treat Duchenne Muscular Dystrophy, but Congress Has Problems
FDA approves first drug for Duchenne muscular dystrophyEven though an FDA panel recommended against approving a new drug to treat Duchenne muscular dystrophy in April, the agency granted accelerated approval to eteplirsen injection (Exondys 51, Sarepta Therapeutics) in mid-September.
Boy complains of acute onset twitchingAn 8-year-old boy presents to the emergency department (ED) with acute onset of twitching. The child began experiencing uncontrollable tics involving his bilateral upper extremities and shoulders 2 hours prior to presentation, with no associated loss of consciousness.
New guidance issued for CMD managementClinicians caring for children with congenital muscular dystrophy (CMD) now have new evidence-based recommendations to guide their evaluation, diagnosis, and management of the disease in pediatric patients.
Early clues to muscular dystrophy in boysA combination of 2 developmental delays in toddler boys can alert physicians to the possibility of Duchenne muscular dystrophy and lead to earlier diagnosis, a new study reports.