Medpage Today on MSN3d
First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise
The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...
FDA approves tablet form of Evrysdi for spinal muscular atrophy
The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for ...
pharmaphorum2d
SMA treatment with Evrysdi 'could start prenatally'
A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...
Monthly Prescribing Reference4d
New Tablet Formulation of SMA Therapy Evrysdi Gets FDA Approval
The tablet formulation can either be swallowed whole or dispersed in a teaspoon of filtered (non-chlorinated) water.
Live Science on MSN1d
Deadly motor-neuron disease treated in the womb in world 1st
An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...
Royal Bank of Canada Forecasts Strong Price Appreciation for PTC Therapeutics (NASDAQ:PTCT) Stock
PTC Therapeutics (NASDAQ:PTCT – Get Free Report) had its price objective hoisted by Royal Bank of Canada from $60.00 to $63.00 in a research report issued to clients and investors on Tuesday,Benzinga ...
SMA1d
Child doing well after mother had SMA treatment while pregnant
A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...