Don't just read the dates—add them to your schedule. Download our free 2026 Disability Awareness & Assistive Technology ...

After five years, Nido Biosciences has reached the end of the road, closing its doors after its lead neurological disease ...

Join the community workshop in Thiruvananthapuram to support families affected by Spinal Muscular Atrophy and learn about the condition.

But in the 1990s the virus that causes AIDS turned out to offer a solution. Researchers discovered how to strip away HIV’s destructive genes and repurpose it as a “lentiviral vector”—a tool to deliver ...

For the approximately one-in-twelve Canadians who have a rare disease, more bureaucracy is a matter of life and death ...

"ኤድዋርድ ይህን የአንድ ጊዜ ሕክምና ተጠቃሚ ከሆኑት ከ150 በላይ የተመሳሳይ ችግር ካለባቸው ሕጻናት አንዱ ሲሆን፣ እንዲህ ዓይነቶቹ ብዙ የጤና ችግሮች በሚቀጥሉት ዓመታት በሚኖሩ ፈጣን የሕክምና ...

Betsy Bihn, PSA director, and Donna Clements, PSA coordinator and Northeast Regional Extension associate, accepted the honor ...

Royalty Pharma Evrysdi royalty acquisition completed for $240M. RPRX now owns 100% of the tiered royalty on Roche's SMA drug, ...

A 5-year-old British boy with the rare disease spinal muscular atrophy (SMA) has miraculously learned to walk four years after receiving the world’s most expensive gene therapy. On the 31st, the BBC ...

Megan Willis' son, Edward, was given the gene therapy, Zolgensma, after being diagnosed with spinal muscular atrophy when he ...

Cecilia Giménez, the artist behind the viral Beast Jesus restoration, has died at 94. Her legacy lives on in memes, tourism, ...