Rare Diseases
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Nominate a Colleague for a NORD 2017 Rare Impact Award
News
Sickle cell trait raises exertional rhabdomyolysis risk
Among blacks soldiers, sickle cell trait doesn’t raise the risk of death but does raise the risk of exertional rhabdomyolysis.
Conference Coverage
Children under 6 with factor XIII deficiency had no major bleeds with recombinant product
Key clinical point: A recombinant form of factor XIII was effective at preventing serious bleeding episodes in young children with factor XIII-A...
News
Hemophilia drugs top Medicaid spending per prescription
Medicaid’s three most expensive drugs by spending per prescription were for treatment of hemophilia, with the cost leader coming in at almost $59,...
Conference Coverage
Study identifies important predictors for PC/PGL
Key clinical point: SDHB mutation and tumor size may be better predictors of outcomes in patients with pheochromocytoma and abdominal...
Conference Coverage
Study tracks distant metastatic patterns of Merkel cell carcinoma
Key clinical point: Distant metastases of Merkel cell carcinoma most often involve the lymph nodes, followed by the liver, skin, and bone.
Conference Coverage
Study tracks distant metastatic patterns of Merkel cell carcinoma
Key clinical point: Distant metastases of Merkel cell carcinoma most often involve the lymph nodes, followed by the liver, skin, and bone.
News
Midostaurin cut organ damage in systemic mastocytosis
Key clinical point: Midostaurin helped to resolve organ damage related to mastocytosis. Major finding: In all, 45% of patients had complete...
Conference Coverage
Heat shock protein peptide vaccine appears safe, effective for glioblastoma patients
Key clinical point: A heat shock protein peptide vaccine appears safe and effective for patients with glioblastoma in an early stage trial. Major...
Conference Coverage
Mogamulizumab achieves objective responses in relapsed/refractory adult T-cell leukemia-lymphoma
Key clinical point: The anti-CCR4 monoclonal antibody mogamulizumab was superior to other investigator-selected therapies for the treatment of...
Conference Coverage
Temozolomide + RT boosts survival in elderly with glioblastoma
Key clinical point: Combining temozolomide with radiotherapy prolongs survival for elderly with glioblastoma. Major finding: Combining...