Author Archives: Steve Bryson PhD

Antisense Therapy Safely Dampens Mutant C9orf72 in ALS Patient

An experimental antisense oligonucleotide that works to suppress the mutant C9orf72 gene — a cause of amyotrophic lateral sclerosis (ALS) — safely lowered the production of damaging proteins and other molecules in a patient in a pilot trial. “While other teams have documented that this gene can be suppressed in cells…

FDA Grants Priority Review to ALS Therapy AMX0035

The U.S. Food and Drug Administration (FDA) has accepted for review Amylyx Pharmaceuticals‘ application seeking approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The new drug application (NDA) also was granted priority review by the regulatory agency, which reduces review time from the standard…

Combined Gene Therapy Shows Promise, but Not Synergy, in Mice

A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…

Study of Perampanel for ALS Stopped Due to Adverse Events

A small open-label study evaluating the epilepsy medication perampanel in adults with amyotrophic lateral sclerosis (ALS) was halted due to adverse events that affected behavior. Despite the findings, larger clinical trials investigating perampanel in ALS patients are ongoing, which might determine if the medication contributed to behavioral side effects. The…

Amylyx Seeks U.S. Approval of AMX0035 With FDA Filing

Amylyx Pharmaceuticals is seeking approval in the U.S. for AMX0035, its investigational therapy for slowing functional decline in people with amyotrophic lateral sclerosis (ALS). The company submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for regulatory review. The decision had been…

SBT-272 Supports Health of Mitochondria in ALS Mouse Model

An investigational treatment for amyotrophic lateral sclerosis (ALS), SBT-272 was found to sustainably reach different brain regions, and to protect mitochondria — a cell’s energy source — from TDP-43 toxic aggregates in a mouse model of the disease. “We are excited about the promise of SBT-272 as a potential therapeutic…