In a case that was billed as profits versus patients, India’s Supreme Court dismissed a petition in which Roche sought to ...
Irish Mirror on MSN
'The fact it’s not being used is wrong': Parents issue plea to HSE over life-changing ...
The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...
Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...
Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...
Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights the current FDA-approved therapies for spinal muscular atrophy.
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights recent advancements and ongoing clinical trials in the spinal ...
Spinal muscular atrophy affects all the body’s muscles. For a long time, it was considered a disease caused solely by the loss of nerve cells in the spinal cord. Now, a research team at Leipzig ...
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 ...
Comprehensive intervention involving exercise, nutrition, and pharmacological therapy may be warranted for managing muscular atrophy in COPD. Muscular atrophy in patients with chronic obstructive ...
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