Marta Figueiredo, PhD,  managing science editor—

BrainStorm Cell Therapeutics announced that a new cleanroom facility for manufacturing NurOwn, its experimental cell-based therapy for amyotrophic lateral sclerosis (ALS), has received good manufacturing practice (GMP) certification from the Israel Ministry of Health. GMPs are standards set to ensure that batches of a medicine are produced with consistent high…

A subsidiary of Sperogenix Therapeutics has acquired exclusive rights to develop and commercialize ABSK021, an investigational oral therapy for amyotrophic lateral sclerosis (ALS) and other rare neurological diseases in mainland China, Hong Kong, and Macao. Under the terms of the agreement between Sperogenix MedTech and Abbisko Therapeutics, the therapy’s…

AB Science’s proposed measures to ensure patient safety in clinical trials of its experimental oral therapy masitinib were deemed sufficient by the French National Agency for Medicines and Health Products Safety (ANSM), and patient enrollment in the country may resume. The announcement comes less than two months after…

Ubiquitination — the cell’s process of tagging proteins, often to degrade unwanted ones — is essential for the disassembly of stress granules that, by not being cleared, damage cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, a study showed. The study, “Ubiquitination is essential for…

The first part of Biogen’s early access program for tofersen is now open to patients with the most rapidly progressing cases of familial amyotrophic lateral sclerosis (ALS), the company announced. These patients, with familial ALS caused by mutations in the SOD1 gene, will be the first to…

Exservan, the first oral film formulation of riluzole for treating amyotrophic lateral sclerosis (ALS), has been launched in the United States, its developer, Mitsubishi Tanabe Pharma America (MTPA), has announced. Rapidly dissolved when placed on top of the tongue, Exservan — specifically developed to help meet the needs…

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…

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…

Most unpaid caregivers in the U.K. have not had any breaks from their caregiving role during the COVID-19 pandemic, with nearly three-quarters reporting feeling exhausted, stressed, and anxious, according to a survey of more than 2,800 caregivers conducted by Carers UK. During the pandemic, carers lost a mean…

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,…