Grants made possible by the “Million Dollar Bike Ride” and rare disease patient organizations are now available through the University of Pennsylvania Orphan Disease Center. September 16 is the deadline for letters of intent.
The diseases for which research funding is available are: adult polyglucosan body disease; ataxia-telangiectasia; Castleman disease; CFTR nonsense mutations; congenital hyperinsulinism; congenital muscular dystrophy; CRB1 degenerative retinal disease; nonsense mutations in cystic fibrosis; dyskeratosis congenital & telomere biology disorders; fibrous dysplasia/McCune Albright syndrome; generalized lymphatic anomaly and Gorham-Stout disease; Glut 1 deficiency; lymphangioleiomyomatosis; mucolipidosis type IV; mucopolysaccharidoses; Niemann Pick type C; Pitt Hopkins syndrome; RASopathies; Tay-Sachs, Sandhoff, GM1, or Canavan disease; and Snyder-Robinson syndrome.