Thirty-million Americans live with a rare disease, with half diagnosed being children. Despite the serious challenges facing ...

AscellaHealth has introduced its latest Specialty & Rare Pipeline Digest™, a quarterly publication designed to provide ...

Florida leaders can enact a new law, the Right to Try for Individualized Treatments, that allows Floridians with rare ...

The sudden resignation of Peter Marks, M.D., Ph.D., from the FDA branch that regulates biological products for humans ...

Patient-reported outcomes measures in generalized myasthenia gravis (gMG) are more important than ever, for both those ...

After some high-profile crashes, the one-time biotech darling is inching toward success with its Hunter syndrome treatment, ...

Biomarker testing also extends beyond cancer care. The science is being used in the treatment of other diseases including ...

AI helped Thomas Wagner understand Alexander disease, find connection with other conditions and discover researchers who ...

Initial pediatric subject shows sustained benefit with QRX003; second patient approved for full-body treatment in ongoing ...

CHICAGO (WLS) -- A case of tuberculosis was reported in a north suburban high school. The Lake County Health department said ...

Immunodeficiency disorders linked to cytoskeleton defects are rare and complex, often presenting with diverse clinical features.

At only two days old, Sophie was losing too much weight, and too quicky.