Building awareness and communities to support and find treatments for Rare Diseases
Building awareness and communities to support and find treatments for Rare Diseases
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
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
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
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
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
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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