The Canadian Rett Syndrome Coalition - comprising the Rett Syndrome Society of British Columbia (RSSBC), Rett Syndrome Society of Alberta (RSSA), Saskatchewan Rett Syndrome Association (SRSA), ...

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

So these studies include the [emugrobart] data in SMA and FSDH and the CT-868 data in type 1 diabetes and the CT-996 data ... spikes in diabetic sugar measurements as well as exacerbations of ...

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 ...

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 ...

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 ...