Tonic motor activation therapy was associated with reductions in RLS severity, along with tolerable reductions in dopamine agonist dosing, in adults with medication-refractory RLS.
The FDA lifted a clinical hold on simufilam, clearing Filana Therapeutics, formerly Cassava Sciences, to begin a phase 2a study in TSC-related epilepsy.
The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.
Marketed as Fayuvi, the 1-time AAV9 gene therapy showed sustained cerebrospinal fluid heparan sulfate drops and developmental gains among pediatric patients with mucopolysaccharidosis type IIIA in prior studies.
Amy Waldman, MD, Medical Director of the Leukodystrophy Center at Children's Hospital of Philadelphia, discusses zilganersen's mechanism, safety profile, clinical considerations, and what the first-ever approval for Alexander disease means for the broader leukodystrophy field.
MS Compass: Guiding Clinical Care, a podcast hosted by neuroimmunologist Lindsay Ross, MD, brings you an exclusive interview with Rachel Galioto, PhD. [LISTEN TIME: 33 minutes]