The EveryLife Foundation for Rare Diseases Marks 15th Annual Rare Disease Week on Capitol Hill

For the 15th consecutive year, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) will welcome advocates from ...
ALS News Today

How ALS patients can show their stripes for Rare Disease Month

Columnist Dagmar Munn encourages "zebras" to show their stripes in February as part of Rare Disease Month and Rare Disease ...

Next frontiers of rare disease research

Scientific advances and cutting-edge technologies are reshaping how rare diseases are diagnosed and treated, bringing new ...

Rare Disease Month: GENA Advances Early Detection and Diagnosis Through AI-Driven Genomic Intelligence

GENA highlights how AI and genomic intelligence transform rare disease identification, speeding diagnosis and improving ...
BioProcess International

FDA rare disease fast-track program passes Senate in big win for small biotechs

The milestone is being celebrated as a “huge moment” for rare disease drug development.

'Shaped my family’s life': Your rare disease stories

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare ...
News Medical on MSN

Advanced use of MRI aids in accurate identification of rare, atypical parkinsonian disorders

An international study led by researchers from the Sant Pau Research Institute (IR Sant Pau) shows that advanced use of ...
Fierce Biotech

FDA rejects Disc's rare disease drug despite commissioner's voucher

The FDA has rejected a rare blood disease candidate from Disc Medicine, a compound that been granted expedited review through ...
News9Live on MSN

One gene, many roles: How DNA makes rare diseases look different in every patient

Advances in DNA sequencing have revealed something surprising—not everyone with the same genetic change develops the same ...
InvestorsHub on MSN

Spruce Biosciences reports positive FDA meetings as it prepares TA-ERT filing for rare pediatric disorder

Spruce Biosciences, Inc. (NASDAQ:SPRB) said it has completed two Type B meetings with the U.S. Food and Drug Administration (FDA), clearing key regulatory discussions ahead of its planned biologics ...

TrumpRx must work for families with rare diseases | Opinion

The author's son has hemophilia, a rare disorder requiring expensive, life-saving medication. The TrumpRx program aims to ...

Rare Disease Day

Wisconsin Live Desk talks with the founder of EverythingALS ahead of rare disease day. Kate Spade Outlet has slashed a $359 ...