The FDA designation expands Atossa’s investigational therapy into rare pediatric neuromuscular disease. ・The program could become eligible for a Priority Review Voucher upon future approval.

HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...

The European Union has recommended for marketing authorization of AstraZeneca Plc's AZN Soliris (eculizumab) for expanded use in refractory generalized myasthenia gravis (gMG) in children and ...

Neurological disorders affect the function of the nervous system. Common pediatric neurological disorders include ADHD, autism spectrum disorder, epilepsy, and cerebral palsy. Neurological disorders ...

Oregon Health & Science University’s Doernbecher Children’s Hospital in Portland has received a $16 million gift to establish a clinic for pediatric neuromuscular disorders. 1. The $16 million ...

Designation expands (Z)-Endoxifen program into rare pediatric neuromuscular disease and may qualify Atossa for a future Priority Review Voucher upon approval SEATTLE, Dec. 11, 2025 /PRNewswire/ -- ...

(Wedgefield) July 3, 2006 - For some kids in Sumter County, summer fun means heading to camp. Twenty-two children with neuromuscular diseases are at Camp Burnt Gin in Wedgefield for a weeklong camp.

The National Organization for Rare Disorders (NORD(R)) today urged Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program by year-end, warning that continued ...

Jonathan Strober, MD, discusses advancements in pediatric myasthenia gravis, emphasizing the need for better treatment standards and effective patient care strategies.

Richard S. Finkel, MD, director, St. Jude Center for Experimental Neurotherapeutics, has been named to Time Magazine’s TIME100 Health list for 2025. TIME has named Richard S. Finkel, MD, Center for ...