Highlights advancing clinical-stage pipeline, including two major value-creating programs for primary immunodeficiencies ...
GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...
4don MSN
This 11-year-old has battled a rare disease for years — a breakthrough drug helped deliver relief
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...
The EveryLife Foundation for Rare Diseases (EveryLife Foundation) announced the awardees for the 2025 RareVoice Awards, which ...
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare pediatric disease priority review voucher program, among other initiatives, ...
This Rare Disease Day, observed on Feb. 28 worldwide, the American Kidney Fund is committed to improving the understanding of ...
Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.
Patient groups argue that customs exemptions for rare disease treatments offer minimal relief without sustained government ...
In the Union Budget 2026, the government announced targeted relief for cancer patients by exempting basic customs duty on 17 ...
Sanofi will take venglustat to regulators for Gaucher disease but an application for Fabry disease is less clear after the failure of a Phase III trial.
The Orphan Drug Act revolutionized rare disease treatment, yet rising costs and access issues challenge equitable health care solutions.
Muscular Dystrophy Association applauds bipartisan Congressional passage of funding and policy priorities advancing research, access to care, and treatments for neuromuscular diseases. Photo L to R: ...
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