The first patient has been dosed in a Phase 2 clinical trial testing RAG-17 in people with ALS who carry mutations in the ...

Ractigen Therapeutics is pleased to announce that the first patient has been dosed in the Phase II clinical trial of RAG-17, an innovative siRNA therapy targeting SOD1-mutated amyotrophic lateral ...

Amyotrophic lateral sclerosis (ALS) remains one of the most devastating and biologically elusive neurodegenerative diseases. Despite decades of research, its underlying mechanisms are still not fully ...

Subset analysis of patients whose ALS did not progress from a long-term survival study showed NP001 saved lung function and extended life by 22 months vs. a control group (~70% on riluzole) NP001 ...

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.

Some patients with a rare form of ALS benefited from an experimental therapy, with biomarker evidence of reduced injury to neurons and even limited functional recovery. When Columbia neurologist and ...

A new drug may slow progression of -- and even reverse -- symptoms of a rare form of amyotrophic lateral sclerosis, or ALS, a new study published Monday finds. The drug, tofersen, targets a very ...

Typically, the goal of ALS treatments is to slow the disease or halt progression. But new data from the antisense oligonucleotide (ASO) jacifusen for FUS-ALS show that two patients went beyond the ...

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a neurodegenerative disease that affects the neurons in the brain and spinal cord. In the United States alone there are ...

Jonathan Wosen is STAT’s West Coast biotech & life sciences reporter. You can reach Jonathan on Signal at jwosen.27. Researchers have found that patients with amyotrophic lateral sclerosis have ...

A new drug may slow progression of — and even reverse — symptoms of a rare form of amyotrophic lateral sclerosis, or ALS, a new study published Monday finds. The drug, tofersen, targets a very ...