Kevin Schaefer hadn’t been in an airport since he was 4 years old, so he had been looking forward to flying from his home in Cary, North Carolina, to Anaheim, California, in June for the 2019 Cure SMA Conference. As it turned out, his experience didn’t go as expected.
AI Therapeutics‘ lymphoma candidate LAM-002 was identified by the company’s artificial intelligence-based platform — called Guardian Angel — as a possible treatment for amyotrophic lateral sclerosis (ALS). Researchers from AI have already tested and confirmed LAM-002’s therapeutic potential in an ALS context. Now, an independent…
A $1 million gift from the Manouk Djoukhadjian Family Foundation II will create Quebec’s first philanthropic research chair on amyotrophic lateral sclerosis (ALS). The donation to the Armand-Frappier Foundation will establish the Anna Sforza Djoukhadjian Philanthropic Research Chair to advance ALS research at the Institut national…
Stealth BioTherapeutics announced the initiation of a Phase 1 trial to assess the safety and tolerability of its investigational oral treatment, SBT-272, in healthy volunteers. SBT-272 is being developed to treat neurodegenerative diseases marked by problems in the workings of mitochondria (a cell’s powerhouse), including amyotrophic lateral sclerosis…
The U.S. Food and Drug Administration (FDA) has authorized the evaluation of the first three candidate therapies for amyotrophic lateral sclerosis (ALS) in the HEALEY ALS Platform Trial. This trial model, designed to accelerate the development of ALS therapies by assessing multiple treatment candidates simultaneously, is the first of…
Athletes who played professional football — known as soccer in the U.S. — are more than three times more likely to die of a neurodegenerative disease than are the general population, a study from Scotland has found. Its researchers compared causes of death over 18 years for about 7,700 former professional…
A first patient has been dosed in a Phase 2 trial testing a nanomedicine called CNM-Au8 as a potentially disease-modifying therapy for people with amyotrophic lateral sclerosis (ALS). This safety and efficacy study, due to finish in April 2021, is also completely enrolled, Clene Nanomedicine, the treatment’s developer, announced…
A single spinal injection of an investigational RNA therapy blocked motor neuron degeneration and saved motor function in animal models of familial amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations, a study  reports. Giving the treatment — a virus-delivered gene-silencer targeting the SOD1 gene —  before the onset of…
A recently discovered inflammatory mechanism triggered by Schwann cells —  specialized cells that produce the protective myelin sheath around neurons —  in amyotrophic lateral sclerosis (ALS) can be alleviated with masitinib, a study in rat models shows. The study, “Schwann cells orchestrate peripheral nerve…
To help accelerate development of effective treatments for amyotrophic lateral sclerosis (ALS), the ALS Association is investing $3 million in the disease’s first platform trial. The award is $1 million annually for three years to support the platform investigation, a type of clinical study that evaluates the…
Get regular updates to your inbox.