NIH rare disease program selects third round of projects

NIH selected four preclinical Eli Lilly and Co. (NYSE:LLY) to treat hypoparathyroidism and a project from Beth Israel Deaconess Medical Center to use rapamycin to treat hypertrophic cardiomyopathy in Leopard syndrome, which is caused by protein tyrosine phosphatase non-receptor type 11 ( PTPN11) mutations. The third and fourth projects are focused on treating retinitis pigmentosa and include a small molecule pharmacological chaperone from Bikam Pharmaceuticals Inc. (Alachua, Fla.) and retinal-derived progenitor cells from the University of California, Irvine. ...