Amylyx Pharmaceuticals is facing a new hurdle — a second round of questions from a European Medicines Agency (EMA) committee — in its quest to have AMX0035 approved as a treatment for amyotrophic lateral sclerosis (ALS) in Europe. The questions, from the Committee for Medicinal Products for…
News
Anew Medical‘s targeted gene therapy candidate, ANEW-202, eased multiple mechanisms associated with amyotrophic lateral sclerosis (ALS), leading to improved muscle function and survival in a mouse model of the disease. The gene therapy, licensed on an exclusive worldwide basis from the Autonomous University of Barcelona, in Spain, is…
QurAlis has secured $88 million to advance the clinical development of QRL-101 and QRL-201, its lead candidates for amyotrophic lateral sclerosis (ALS). The new series B financing, a second round of funding after the company met certain research milestones, brings the total investment to $143.5 million. The proceeds will…
Radicava ORS, an oral formulation of Radicava (edaravone), is now available to U.S. veterans living with amyotrophic lateral sclerosis (ALS), the therapy’s developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The therapy was added to the U.S. Department of Veterans Affairs National Formulary (VANF) in June 2022, shortly after its…
The Muscular Dystrophy Association (MDA) is hosting its 16th annual MDA Atlanta Night of Hope Gala: A Benefit for ALS Research to raise funds for amyotrophic lateral sclerosis (ALS) research and care. The event will be held 6-11 p.m. EDT March 25 at the InterContinental Buckhead Atlanta. It…
Renowned neurologist and amyotrophic lateral sclerosis (ALS) investigator Merit Cudkowicz, MD, of Massachusetts General Hospital has been named this year’s recipient of the Muscular Dystrophy Association (MDA) Legacy Award for Achievement in Clinical Research. The award, which recognizes outstanding accomplishments in neuromuscular research, will be presented March 20…
Treatment with CNM-Au8 significantly delayed clinical worsening in people with amyotrophic lateral sclerosis (ALS), according to new exploratory analyses from the therapy’s arm of the HEALEY ALS platform trial. The experimental therapy, from Clene Nanomedicine, had previously been found to reduce the risk of death by more…
AviadoBio has entered into a worldwide exclusive agreement to develop and commercialize Neurgain Technologies’ new technique to deliver gene therapies for diseases that affect the spinal cord. The approach will be used at first to deliver AviadoBio’s investigational gene therapies for amyotrophic lateral sclerosis (ALS). It involves…
RAG-17, an investigational therapy from Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to medicines intended to treat life-threatening or chronically debilitating diseases that affect fewer than 200,000 individuals…
Amyotrophic lateral sclerosis (ALS) patients treated earlier with CNM-Au8 in a clinical trial experienced a significantly slower disease progression than those who started treatment nine months later. These latest findings from the RESCUE-ALS Phase 2 trial (NCT04098406) and its open-label extension (OLE) study (NCT05299658) add to earlier reports that…
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