The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...
The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).
Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...
Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve ...
Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...
Doctors diagnosed Carmen Lopez with spinal muscle atrophy (SMA) at birth, a rare genetic disorder that affects motor neurons ...
On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...
Basel: Roche, announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an ...
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