pharmaphorum11h
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 ...
Ionis Continues to Expand Its Cardiology and Neurology Portfolio, Supporting Narrow Moat
Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...
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 ...
Hosted on MSN10h
Spinal Muscle Atrophy: Types, Symptoms, Treatment, Prevention
Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...
News Medical on MSN8h
Promising results from first prenatal therapy for spinal muscular atrophy
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...
ENDPOINTS NEWS6h
In sin­gle case, spinal mus­cu­lar at­ro­phy pill giv­en be­fore birth spared ba­by of ge­net­ic dis­ease symp­toms
Mother took Evrysdi for SMA before birth; baby later received Zolgensma. Dr. Richard Finkel at St. Jude led study showing no ...
Nature22h
Rare genetic disorder treated in womb for the first time
A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...
UPMC-Pitt trial offers hope for patients with rare neurodegenerative diseases
Doctors diagnosed Carmen Lopez with spinal muscle atrophy (SMA) at birth, a rare genetic disorder that affects motor neurons ...