Alice was doubled over in agony again, as she was rushed into hospital with that familiar griping pain and vomiting. Many Doctors would be unfamiliar with the illness, Superior Mesenteric Artery ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New ...
Spinal muscular atrophy is a disease that most often strikes babies and young children, making it hard for them to move their muscles. SMA is passed down in families from parents to their children. It ...
Type 1 spinal muscular atrophy (SMA) is a progressive neuromuscular disease characterized by an onset at 6 months of age or younger, an inability to sit without support, and deficient levels of ...
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA). Among SMA ...
An expert neurologist, Emma Ciafaloni, MD, provides an overview of the epidemiology of spinal muscular atrophy and discusses symptom burden, differential diagnosis, and prevalence. Dennis Scanlon, PhD ...
Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile, with ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback