GENA highlights how AI and genomic intelligence transform rare disease identification, speeding diagnosis and improving ...
The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...
Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare ...
Rare lung diseases affect people nationwide, yet many patients go undiagnosed or misdiagnosed for years. One rare lung disease that illustrates the challenges of timely diagnosis is autoimmune pulmona ...
Researchers have developed a single genome-editing strategy, known as PERT, that could potentially treat various diseases caused by nonsense mutations.
Even when promising clinical trials exist, and the FDA allows expanded access, rare disease patients like my granddaughter ...
The FDA in October 2025 granted bitopertin its Commissioner’s National Priority Voucher but after a shortened review time has decided the data did not support regulatory approval for treating ...
This Rare Disease Day, observed on Feb. 28 worldwide, the American Kidney Fund is committed to improving the understanding of rare kidney diseases by providing educational resources.
Ventavis® is an established inhaled prostacyclin analogue for the treatment of primary pulmonary hypertensionAcquisition reinforces Essential Pharma’s mission to serve the needs of rare disease ...
Wisconsin Live Desk talks with the founder of EverythingALS ahead of rare disease day. Kate Spade Outlet has slashed a $359 ...
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