The MESA data show stabilization of function in participants on sevasemten versus the predicted functional decline observed ...

The US regulator has not, however, cleared leucovorin for autism spectrum disorder (ASD) itself, stepping back from the furore that broke out last year when President Trump claimed it could be a ...

Barclays 28th Annual Global Healthcare Conference March 11, 2026 8:00 AM EDTCompany ParticipantsMatthew Klein - CEO ...

People with BMD who received sevasemten in clinical trials maintained stable motor function over several years of follow-up, new data show.

PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...

Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other rare disease indications with ...

New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...

Exposure to wildfire smoke can more than double the risk of respiratory failure or death for ALS patients, a study found.

Insmed has launched a first-in-human Phase 1 trial testing its experimental ALS gene therapy INS1202 in people with the disease.

This week's biotech landscape witnessed key FDA approvals, EU approvals, IND clearances, acquisitions, and clinical trial data readouts across key therapeutic areas such ...

BACKGROUND: Peripheral artery disease (PAD) and its severe form, chronic limb-threatening ischemia (CLTI), significantly impair blood flow to the lower extremities, affecting millions of adults ...