Showing 4187 results for "als"

Sporadic ALS

About 90% of cases of amyotrophic lateral sclerosis (ALS), the progressive neurological disorder also known as Lou Gehrig's disease, are characterized as sporadic ALS, meaning they occur spontaneously. The term sporadic in ALS means without a clear pattern, and the individuals with these cases have no known family history of the disease.

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…

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…

Types of ALS

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that can be classified into different types based on factors like genetics, a patient's family history, and the disorder's early symptoms. Across all types, ALS is marked by the loss of motor neurons...

FDA names RAG-17, targeting ALS gene mutations, an orphan drug

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…