Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

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

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.

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.