In The Spotlight

Autism Spectrum Disorder: Progress Toward Earlier Diagnosis

Researchers Aim to Repurpose Former Experimental Cancer Therapy to Treat Muscular Dystrophy

The XVIth International Symposium on Amyloidosis

New Research Projects on Pearson Syndrome Underway

NIH, Academia and Patient Advocate Collaboration Speeds Niemann-Pick Type C1 Research

Unprecedented Trans-NCATS Collaboration Enables Rapid Advancement of Rare Lung Disease Therapy to Human Trials

NIH Program Strives to Turn More Lab Discoveries Into Real-World Treatments

Funding Opportunities California Institute for Regenerative Medicine (CIRM), California's Stem Cell Agency

NHGRI Partners With Patient to Test New Proteus Syndrome Treatment

Study Reveals Promising Results for Treating Methylmalonic Acidemia