Three-year-old Meher from Noida was diagnosed with Turner Syndrome at PGICH using QF-PCR, a first for the institute. Early ...

Unresolved compliance issues at a manufacturing plant operated by Novo Nordisk have led to a complete response letter for Scholar Rock's lead drug for spinal muscular atrophy (SMA). The CRL is related ...

A big part of Biogen's efforts to defend its spinal muscular atrophy (SMA) business has been dealt a setback by the FDA, which declined to approve a high-dose version of its flagship Spinraza product.

2025年10月，专注于RNA靶向药物研发的Ionis ...

NORTHAMPTON, MA / ACCESS Newswire / October 20, 2025 / Originally published on Aflac NewsroomIn a quiet corner of the Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, somet ...

The 2022 Option Agreement between Astellas and Taysha has expired Regaining full rights to TSHA-102 Rett syndrome program enables Taysha to focus on driving its long-term value with full strategic fle ...