In addition to their suffering, rare disease patients often have to face the harsh reality that few pharmaceutical companies will ever be able to offer new treatments for their condition because the costs of new treatments will never be recovered from such a small market. But there are ways they can be helped.
The U.S. Food and Drug Administration’s “Orphan Drug Designation” offers a wide range of benefits that help organizations developing treatments for diseases and conditions affecting fewer than 200,000 patients in the United States. It was recently granted to McGill University for research conducted at the MUHC into the use of the drug fenretinide for the treatment of pulmonary infections caused by Pseudomonas aeruginosa in patients with Cystic Fibrosis (CF).
“We strongly believe that fenretinide has the potential to improve morbidity and prognosis in CF patients at different levels,” said Dr. Danuta Radzioch, a professor of Experimental Medicine at the MUHC and McGill’s Faculty of Medicine.
Read complete McGill press release.
Agence Science Presse