Abu Dhabi’s Sheikh Khalifa Medical City has become the first hospital globally to administer ITVISMA, a one-time gene therapy ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A person with this disorder will feel weak in some parts of the body, especially ...

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 ...

Living with a rare disease can be incredibly isolating, not just for those affected but for their families as well. For individuals with Spinal Muscular Atrophy (SMA), a genetic condition that weakens ...

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

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

WASHINGTON — Three people with a muscle-destroying disease destined to worsen got a little stronger – able to stand and walk more easily – when an implanted device zapped their spinal cord. On ...

MRI and ultrasound techniques can be used to diagnose the condition; but the former is not best suited to the examination of young children, and the latter cannot detect specific biomarkers of ...

A leading Abu Dhabi hospital has become the first in the world to deliver a pioneering gene-therapy treatment that is poised ...

Mumbai: Meet Khushi Kumari Yadav, a 20-month-old little warrior diagnosed with Spinal Muscular Atrophy (SMA) Type 2. Despite being a cheerful and curious toddler, Khushi faces daily struggles due to ...

Investing.com -- Royalty Pharma Plc (NASDAQ:RPRX) stock gained 1.5% in after-hours trading Monday following the company’s announcement that it has acquired the final portion of PTC Therapeutics ’ ...