Our inbox has been flooded with emails from viewers and readers who want to share their personal stories — people who want to ...
The author's son has hemophilia, a rare disorder requiring expensive, life-saving medication. The TrumpRx program aims to ...
The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...
Among them was David Liu, a biochemist at the Broad Institute and Harvard University in Cambridge, Massachusetts. Liu isn’t a ...
Feb 9 (Reuters) - Regenxbio said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for ...
This Rare Disease Day, observed on Feb. 28 worldwide, the American Kidney Fund is committed to improving the understanding of ...
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Defective fat cells drive metabolic disease in lipodystrophy
Many people may have a dim view of their fat tissue, yet scientists have come to recognize adipose as a necessary and ...
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Millions of children with rare diseases set to benefit from faster diagnosis and better treatments
A new platform is being developed by Great Ormond Street Hospital and LifeArc in collaboration with the Children’s Hospital Alliance ...
Wisconsin Live Desk talks with the founder of EverythingALS ahead of rare disease day. Kate Spade Outlet has slashed a $359 ...
The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding ...
The FDA has rejected Regenxbio’s biologics license application for RGX-121, an experimental gene therapy for Hunter syndrome. In a complete response letter issued Feb. 7, the FDA cited concerns about ...
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