Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

Medcare Royal Speciality Hospital (MRSH) in Dubai has become the world’s first hospital outside the USA, to offer a newly ...

"It's encouraging and it gives me hope that they're finally understanding that we deserve to have treatment just like the rest and that we deserve a chance," she said. "At least a ...

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 ...

Patients with Spinal Muscular Atrophy urge the Prime Minister for access to affordable generic Risdiplam to save lives.

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

Ashley Webb, MD, Program Director, Pediatric Neurology, at the University of Texas Health Science Center at Houston McGovern Medical School, discusses the need to make genetic therapy more accessible ...

Former Little Mix star Jesy Nelson has revealed her twin daughters, Ocean Jade and Story Monroe Nelson-Foster, have been diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic condition ...

Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...