Children’s Hospital of Pittsburgh Foundation received 2 grants totaling $2.5 million, from The Legacy of Angels Foundation to support the diagnosis, treatment, and research of rare and devastating neurological diseases.
News from NORD and its Member Organizations
Cushing disease is a condition characterized by an excess of the cortisol in the blood level cause by a pituitary tumor secreting adrenocorticotropic hormone.
This week in JAMA, clinicians in Brazil report on the surprisingly high rate of ocular abnormalities observed in babies born with microcephaly
Acquired thrombotic thrombocytopenic purpura (aTTP) is an ultra-rare, autoimmune blood clotting disorder.
Amarantus has received orphan drug designation for eltoprazine in the treatment of Parkinson's disease levodopa-induced dyskinesia.
On February 8, the FDA put a partial clinical hold on CTI BioPharma’s clinical study of pacritinib after patients died of intracranial hemorrhage, cardiac failure and cardiac arrest.
Medikidz Explain Urea Cycle Disorders, created in collaboration with Horizon Pharmaceuticals, tells the story of Kyle, who is a young boy living with UCD.
Are you Phelan lucky for St Patrick’s Day?