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 ...
Live Science on MSN22h
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 ...
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.
Barchart on MSN3d
2 Hot Growth Stocks Poised for Gains of Up to 370%, According to Wall Street
While investing in biotech stocks can be risky due to clinical trial failures and regulatory hurdles, successful drug approvals can result in massive stock gains. Let’s look at two such growth stocks ...
MedPage Today3d
First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise
The child was treated in utero with risdiplam (Evrysdi), which is approved to treat SMA in pediatric and adult patients, and continues to receive the oral drug daily. SMA is caused by reduced ...
SMA23h
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 ...
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 ...