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

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 ...

More than 2 years after the child was born, no identifiable features of SMA have been observed, reported Richard Finkel, MD, of St. Jude Children's Research Hospital in Memphis, Tennessee, and ...

Finding the right pair of shoes can be a challenge for many, but for Aaron Warren, it was a personal struggle that led to a ...

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 ...

MIT biologists have discovered a new type of RNA-splicing regulation that helps to determine which protein-coding exons will be included in messenger RNA transcripts.

About a dozen drugmakers are developing new weight-loss treatments aimed at preserving muscle, and industry analysts, ...

Popular weight-loss therapies like Novo Nordisk's Wegovy and Eli Lilly's Zepbound result in both fat and muscle loss.

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