A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.

Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...

A multidisciplinary panel of experts discusses the clinical burden of spinal muscular atrophy on patients, families, and caregivers and considers the importance of patient education and advocacy.

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

—The slow progression of SMA in adults and older children complicates evaluation of treatment response to newer therapies. Longitudinal quantitative MRI (qMRI) may give clinicians a better handle on ...

India makes many of the world’s drugs, but treatments for rare diseases like spinal muscular atrophy are imported and prohibitively costly. In desperation, parents are raising funds on social media.

DUBAI, United Arab Emirates--(BUSINESS WIRE)--Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a ...

NEW YORK, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has acquired the final portion of PTC Therapeutics’ remaining royalty on Roche’s Evrysdi for $240 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...