A mom was overjoyed when she learned a drug could save her baby's life. Then she learned it cost $2 million.

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...
Staten Island Advance

Understanding Spinal Muscular Atrophy: Help little Aubrey

STATEN ISLAND, N.Y. -- Three-year-old Aubrey Paige Ibrahim is a beautiful little girl fighting a battle most adults would find challenging, if not impossible. Born on May 31, 2013, Aubrey was ...

Facioscapulohumeral Muscular Dystrophy Clinical Trial Pipeline Shows Potential with Active Contributions from 10+ Key Companies | DelveInsight

In November 2025, Epicrispr Biotechnologies Inc. announced a clinical trial is to learn how safe and tolerable EPI-321 is and ...
WFLA News Channel 8

Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community

New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...
Medical Xpress

What you should know about spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
Morningstar

FDA Approves New Tablet Form of Evrysdi for Treatment of Spinal Muscular Atrophy

New York, Feb. 12, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam ...
Healio

Apitegromab linked to improved outcomes for those with spinal muscular atrophy

Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...
Healio

Gene therapy improves motor function in spinal muscular atrophy at 52 weeks

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Patients treated with onasemnogene abeparvovec recorded ...
Yahoo Finance

Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community

Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community The Muscular Dystrophy Association ...
Mena FN

Muscular Dystrophy Association Calls FDA Approval Of Novartis' Itvisma (Onasemnogene Abeparvovec-Brve) A Major Step Forward For The Spinal Muscular Atrophy Community

(MENAFN- GlobeNewsWire - Nasdaq) The only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA). New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- ...