Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...

U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...

Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.

While the leading cause of kidney disease is diabetes, many other factors can lead to kidney disease and failure – including ...

Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

Over the next two years, the planned funding will be allocated to projects that have received some FDA feedback as well as emerging efforts.

GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...

NORD's growing network, now 46 institutions strong, represents a coordinated nationwide effort to strengthen rare disease care and research collaboration With these new members, the network now ...

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

The Rare Disease Researcher Interest Group seminar series brings together researchers from different fields to learn about and discuss rare disease research, regardless of experience level with rare d ...

KUALA LUMPUR, Feb 4 — More than 12,000 patients in Malaysia are currently receiving treatment for rare diseases ...