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Top Medical News Today

FDA Approves Rydapt (midostaurin) for Rare Blood Cancers
The FDA has approved Rydapt for the treatment of adult patients with newly diagnosed acute myeloid leukemia in combination with chemotherapy.
Harrington Discovery Institute Teams with Takeda for Advancement of Rare Disease Research
Takeda Pharmaceutical and Harrington Discovery Institute announced that they have agreed to a partnership for rare disease treatment and therapeutics research.
FDA Approves BioMarin's Batten Disease Drug. Cost Per Year is $702,000
Approval based on data from a Phase 1/2 study. BioMarin also received a Rare Pediatric Disease Priority Review Voucher which they can use on ANY future drug or sell it to another company.
BREAKING NEWS: Expanded Usage of Stivarga Approved by FDA
Approved usaged of Stivarga (regorafenib) was expanded by the FDA and now includes treatment of patients with hepatocellular carcinoma (HCC or liver cancer).
BREAKING NEWS: Gottlieb Approved by Senate as FDA Nominee
A U.S. Senate panel voted to approve Dr. Scott Gottlieb as the head of the FDA. His nomination will be sent for a confirmation vote.
Progeria Patients Are No Sideshow
Rupesh Kumar, 21-year old of India, has been diagnosed with Progeria. He was once almost purchased and exhibited as a sideshow in the circus.
Eidos Therapeutics Launched by BridgeBio Pharma for Development of TTR Amyloidosis Treatment
BridgeBio Pharma announced the launch of Eidos Therapeutics to develop an original small-molecule treatment named AG10 for transthyretin (TTR) amyloidosis.
Insurance Company Loses Battle with SMA Patient, Must Pay for FDA Approved Drug
Spinraza is indicated for “the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.” The definition, however, has not stopped some insurance companies from trying to limit which SMA patients receive the expensive drug – and at $125,000 per dose, the drug is expensive.
Duchenne Muscular Dystrophy – Cost of Care High, Quality of Studies Low
A systematic review of 58 studies involving Duchenne muscular dystrophy (DMD) by Leadley et al in the Orphanet Journal of Rare Disorders reaffirmed the tremendous cost this rare disease has on a family.
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