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

The programme will focus on accelerating treatments for rare diseases, most of which currently lack approved therapies.

The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding ...

The drug, which has suffered multiple setbacks over the years, could finally reach the market as a treatment for a form of ...

Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...

Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...

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

To be only six years old and have seven breaks and two surgeries, that’s a lot,” Jessica said. “Some days are really hard.” ...

Sanofi’s pipeline features a number of once-hyped candidates that are struggling to deliver in the clinic, and the ...

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 ...