SPOTLIGHT -
What neuropsychiatric conditions are relatives of people with ALS at risk for? Are other genes involved besides C9orf72?
Efgartigimod Effective Across Subgroups, FDA to Hold AdComm Meeting on SRP-9001, Reboxetine Demonstrates Positive Effect on Obstructive Sleep Apnea
Special Episode: Trofinetide Approved for Rett Syndrome
Losmapimod Demonstrates Significant Slowing of Facioscapulohumeral Muscular Dystrophy Over Long-Term Period
Episode 80: Advancing Treatment for Rare Neuromuscular Disorders
Consensus Considerations Developed for SRP-9001 Adverse Event Management in DMD
FDA AdComm on Tofersen Supports NfL as Surrogate End Point of Clinical Benefit, But Is It Enough for Approval?