European approval of AMX0035 for ALS faces new hurdle in review

Anew’s ALS gene therapy shows promise in mice

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 secures $88M to advance ALS therapies

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, oral form of edaravone, available to US veterans

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…

ALS researcher Merit Cudkowicz to receive MDA legacy award

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…

CNM-Au8 found to delay clinical worsening in ALS HEALEY trial

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…