Set to enter larger trial across 5 countries, it works by permanently turning off gene that causes high bad cholesterol ...

Spero Therapeutics and partner GSK are stopping a late-stage trial of an experimental oral drug for complicated urinary tract ...

Learn how a new genetic therapy could transform treatment for rare ALS—keep reading for remarkable patient outcomes.

Rocket Pharmaceuticals, Inc.’s (NASDAQ:RCKT) prospects have taken a significant hit on the company being forced to halt ...

Researchers say a full-scale clinical trial of the therapy could help many patients with a particularly aggressive form of ...

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.

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 ...

An experimental drug known as ulefnersen has shown remarkable efficacy in treating a rare, aggressive form of ALS ...

Right to Try laws are being adopted and expanded throughout the country. In one state, they’ll allow healthy people to access ...

An experimental drug developed at Duke University School of Medicine could offer powerful pain relief without the dangerous ...

EBC-129 is the first made-in-Singapore antibody-drug conjugate (ADC) to enter clinical development. It selectively targets a tumour-specific N-glycosylated epitope on both CEACAM5 and CEACAM6.

When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he ...