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A novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.
California Startup Exploring Novel ALS Discovery Platform
BHV-0223 Expanded Access Program Opened for ALS, as FDA Application Is Prepared
NP001 Fails to Meet Primary, Secondary Endpoints in ALS Trial
Ralph Kern, MD, MHSc: The State of Therapies for ALS
Jeffrey Gudin, MD; Jeffrey Bettinger, PharmD
Zarazuela Zolkipli-Cunningham, MBChB, MRCP; Mary Kay Koenig, MD; Perry Shieh, MD, PhD, FAAN, FANA
James F. Howard Jr, MD, FAAN
Basil T. Darras, MD
Karissa Gable, MD
Anne M. Connolly, MD, FAAN; Amy Moore, PhD
Jessica Oswald, MD, MPH; Jeremy A. Adler, DMSc, PA-C; Amanda Nasser, MS, NP