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

Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.

This rare genetic disorder was just treated in the womb for the first time ever - Without treatment, those with the more ...

On the upside, the first hurdle is the 2025 high of 0.6395 (February 20). The next level to watch is the 100-day Simple Moving Average (SMA) at 0.6425, followed by the weekly top at 0.6549 (November ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

U.S. President Donald Trump's top security adviser called Thursday for all member states of the North Atlantic Treaty ...

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

February is Rare Disease Awareness month. The upcoming week has been declared Rare Disease Awareness week, with Rare Disease ...

Stax Music Academy will mark its anniversary with concerts and educational efforts, including “Soul of America,” a virtual ...

The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for ...

Super Micro's CEO Charles Liang's ambitious 2026 targets help to confirm that the AI growth story remains intact over the ...