Voyager Therapeutics reassesses SOD1-ALS gene therapy program
Voyager Therapeutics will reassess its gene therapy program for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene…
Articles by Patricia Inácio, PhD
Clinical hold lifted from Phase 1 trial of ALS therapy AMX0114
The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1 clinical trial that’s testing…
Dosing begins in Phase 1 trial of potential ALS therapy RAG-17
A Phase 1 clinical trial testing RAG-17, an investigational therapy for people with amyotrophic lateral sclerosis (ALS) carrying mutations in…
Corcept’s dazucorilant fails to slow ALS progression in clinical trial
Dazucorilant, an oral cortisol modulator being developed by Corcept Therapeutics, didn’t significantly slow disease progression in people with…
FDA grants orphan drug status to RAG-21 for treating FUS-ALS
RAG-21, a therapy Ractigen Therapeutics is developing for amyotrophic lateral sclerosis (ALS) linked to mutations in the FUS gene,…
EMA recommends NUZ-001 for orphan drug status in Europe
The European Medicines Agency (EMA) has recommended that monepantel, now named NUZ-001, be granted orphan medicinal product designation to treat…
I AM ALS unveils new campaign aimed at supporting caregivers
This November, I AM ALS, a nonprofit dedicated to helping people — both patients and family members — affected…
ALS respiratory data now available to researchers worldwide
A database integrating long-term respiratory data with natural history data from people with amyotrophic lateral sclerosis (ALS) has been…
RNS60 aids mitochondria health, protects motor neurons in ALS mice
A treatment candidate from Revalesio named RNS60 protected motor neurons from damage in a mouse model of amyotrophic…
Target ALS, Modality.AI partner to study AI in ALS
Modality.AI is teaming up with Target ALS on a clinical study of artificial intelligence (AI) as way to…