Author Archives: Marta Figueiredo PhD

ALS Association Wants More Commitment From the FDA

In a response letter to The ALS Association, the U.S. Food and Drug Administration (FDA) has recognized the unmet therapeutic need of people with amyotrophic lateral sclerosis (ALS) and reaffirmed its commitment to the 2019 ALS clinical trial guidance. However, the agency did not provide any details about how it…

New Cellular Model Used to Study FUS Gene Mutations

Mutations in the FUS gene, a common cause of amyotrophic lateral sclerosis (ALS), impair the regeneration and growth of motor neuron extensions toward muscle cells, according to a study using a new miniaturized human model of neuron-muscle interactions. These deficits, along with the reduced number of nerve-motor connections, were lessened by blocking…

Amylyx Seeks Approval in Canada of Lead Candidate AMX0035

Amylyx Pharmaceuticals is seeking approval, in Canada, of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). This regulatory submission “represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” Joshua Cohen, co-founder, co-CEO,…

Grant Backs CRISPR Therapy Research for Familial ALS

Researchers at the University of Texas at Dallas (UT Dallas) have received an award from the U.S. Army Medical Research Acquisition Activity to advance their preclinical research of an innovative CRISPR-based gene editing approach to treat one of the most common causes of familial amyotrophic lateral sclerosis (ALS). The two-year…

Cytokinetics Renews Partnership With The ALS Association

Cytokinetics has reaffirmed its partnership with The ALS Association in the fight against amyotrophic lateral sclerosis (ALS) by continuing to support events and initiatives throughout 2021. The muscle biology biopharmaceutical company also is working to advance the clinical development of its experimental therapy reldesemtiv, designed to improve…

SOL-257 Gene Therapy Targeting TDP-43 Shows Promise in Mice

SOL-257, an experimental gene therapy developed by SOLA Biosciences, delayed disease progression and prolonged the lifespan of a mouse model of amyotrophic lateral sclerosis (ALS). The gene therapy is designed to reduce the abnormal TDP-43 protein that forms toxic clumps in the cells of ALS patients. “Targeting only…