An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

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 ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...

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 ...

Doctors diagnosed Carmen Lopez with spinal muscle atrophy (SMA) at birth, a rare genetic disorder that affects motor neurons ...

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 ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...