Clinical

Videos

The chief medical officer at Cure SMA spoke to the ongoing work that the organization does in advocating for patients with spinal muscular atrophy, particularly for newborn screening.


Mary Schroth, MD: Cure SMA’s Ongoing Advocacy Initiatives

Articles

This additional application, supported by data from the GWPCARE6 trial, seeks to expand the indication of cannabidiol to include seizures associated with tuberous sclerosis complex.

GW Submits sNDA for CBD in Tuberous Sclerosis Complex

Study data demonstrates that treatment with satralizumab reduces risk of relapses in patients with neuromyelitis optica spectrum disorder.

Satralizumab BLA Accepted by FDA for Treatment of NMOSD

The regulatory agency cited 2 concerns in the complete response letter: the risk of infections related to intravenous infusion ports and renal toxicity.

FDA Rejects Duchenne Muscular Dystrophy Drug Golodirsen, Citing Safety Concerns

The genomic revolution has led to increasing opportunities to address complex neurodevelopmental conditions, taking a genetics-first approach.

Developing Targeted Therapeutics in Angelman Syndrome

The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?

Rare Diseases

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