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Living with Adult Cystic Fibrosis: What Does It Mean?
While the average life expectancy of persons living with CF past childhood is 37, researchers have asserted that babies born in the 2000s will live well into their fifties.
Michael Binks, MD, Vice President for Rare Disease Clinical Research at Pfizer talks about the anti-myostatin antibody, domagrozumab, being developed for Duchenne muscular dystrophy.
NantHealth Inc. and the University of Utah are partnering up to analyze the genomic profiles of 1,000 individuals diagnosed with and or have a history of rare life-threatening diseases in their bloodlines.
At the WFH 2016 World Congress this week, Kearney et al are presenting data on the long term efficacy and safety of recombinant FXIII (rFXIII) in patients with the rare and severe bleeding disorder, congenital factor XIII deficiency.
Isabelle Jordon, Chair of the Rare Disease Foundation talks about the microgrants that organization provides to rare disease researchers.
Durhane Wong-Rieger of the Canadian Organization of Rare Diseases (CORD) discusses the need for improved infrastructure in Canada to encourage more clinical trial research in the country.
New York's first case of a baby being born with the zika virus-related birth defect, microcephaly.
The triple drug study found increased bone mineral density, but no other improvement. A new study is underway with lonafarnib with everolimus.
An investigation is currently underway in Brazil to see if the Culex mosquito can transmit the ZIka virus.
Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. Acquired hemophilia A is a rare autoimmune bleeding disorder that attacks Factor VIII.

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