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 ...
Patients with Spinal Muscular Atrophy urge the Prime Minister for access to affordable generic Risdiplam to save lives.
"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...
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 ...
The FDA approved a risdiplam tablet for SMA patients aged 2+ and over 44 lbs, matching the oral solution's efficacy and safety. Risdiplam, an SMN2 pre-mRNA splicing modifier, enhances SMN protein ...
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 ...
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 ...
Many new gene therapy treatments aren’t as profitable as predicted. But the reasons why don’t quite fit Goldman Sachs’ warning ...
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 ...
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