Building awareness and communities to support and find treatments for Rare Diseases
April 17, 2018 By Daniel S. Levine Leave a Comment
Rare Daily Staff The Institute for Clinical and Economic Review, an independent, nonprofit that analyzes the cost-effectiveness of therapeutics, issued a report that found Roche’s Hemlibra delivered health benefits for patients with hemophilia A with inhibitors and that drug lowers overall costs of treatment compared…Continue Reading
April 16, 2018 By Daniel S. Levine Leave a Comment
Earlier this month, Kaiser Health News launched “Pre$cription for Power,” a database that tracks Big Pharma contributions to patient advocacy organizations along with a report about the corrupting influence of this money. The report found that 14 of the largest pharmaceutical companies provided a total…Continue Reading
April 13, 2018 By Daniel S. Levine Leave a Comment
Before most rare disease patients have a name to give their condition, they go on a diagnostic odyssey than can take years and usually involves multiple specialists. For some patients, the answer never comes. In the absence of a diagnosis, patients face significant challenges getting…Continue Reading
April 17, 2018 By Daniel S. Levine Leave a Comment
Rare Daily Staff The U.S. Food and Drug Administration cleared Intrexon subsidiary Exemplar Genetics’ ExeGen ATM MiniSwine model for commercial use as a research model of ataxia telangiectasia, a rare, inherited, neurodegenerative disease. Mutations in the ataxia telangiectasia gene lead to the multisystemic disorder AT.…Continue Reading
April 17, 2018 By Daniel S. Levine Leave a Comment
Rare Daily Staff The U.S. Food and Drug Administration granted approval to Crysvita, Ultragenyx and Kyowa Kirin International’s treatment of children and adults with X–Linked Hypophosphatemia, a rare and progressive genetic skeletal disorder. People with XLH can experience abnormal bone formation, bone pain, lower than…Continue Reading
April 17, 2018 By Daniel S. Levine Leave a Comment
Rare Daily Staff The U.S. Food and Drug Administration approved Rigel Pharmaceuticals’ Tavalisse for the treatment of adults with chronic immune thrombocytopenia, a rare disorder in which the body’s immune system attacks the body’s own blood platelets. Common symptoms of ITP include excessive bruising, bleeding…Continue Reading
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