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Zebrafish can play a decisive role in clinical interpretation of spinal muscular atrophy

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

Tiny fish helps clinicians avoid multi-million-dollar treatment for babies suspected of having spinal muscular atrophy

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...
News Medical

Nusinersen treatment improves spinal muscular atrophy patients’ walking distance

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...
MedPage Today

First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...
Seeking Alpha

FDA Issues Complete Response Letter (CRL) for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations Identified at Catalent ...

Scholar Rock (SRRK) intends to resubmit the apitegromab Biologics License Application (BLA) upon resolution of Catalent Indiana LLC-related observations CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar ...
EurekAlert!

Spinal muscular atrophy patients’ walking distance improved after Nusinersen treatment

Amsterdam, February 14, 2023 – A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been ...