Orphan Disease Center at Penn Medicine: 2017 Million Dollar Bike Ride Pilot Grant Program (LOI)

Support research related to a rare disease represented in the 2017 Million Dollar Bike Ride. Research Focus Areas:

*Adrenoleukodystrophy (ALD), *Adult Polyglucosan Body Disease (APBD), *Ataxia-Telangiectasia, *BPAN- A Neurodegeneration with Brain Iron Accumulation Disorder, *Castleman Disease, *CDKL5, *Congenital Hyperinsulinism (CHI), *Congenital Muscular Dystrophy (CMD), *CRB1 degenerative retinal disease, *Nonsense Mutations in Cystic Fibrosis, *Dyskeratosis Congenita & Telomere Biology Disorder, *Fibrous Dysplasia/McCune Albright Syndrome, *Generalized Lymphatic Anomaly (GLA; a.k.a. lymphangiomatosis) and *Gorham-Stout Disease (GSD), *Glucose Transporter Deficiency Syndrome (Glut 1DS), *Lymphangioleiomyomatosis (LAM), *Mucolipidosis Type IV (ML4), *Mucopolysaccharidoses (MPS),*Niemann Pick Type C (NPC), *Pitt Hopkins Syndrome (PTHS), *RASopathies, *Snyder-Robinson Syndrome, *Tay-Sachs, *Sandhoff, GM-1, or *Canavan Disease.

Funding: Varies depending on the disease. Overhead 10%

http://www.med.upenn.edu/orphandisease/rare-disease-overview.html

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