Rare Diseases
News
FDA approves ravulizumab for treatment of paroxysmal nocturnal hemoglobinuria
The drug’s novel formulation means that patients can be treated every eight weeks, instead of every two weeks as is the case with the previously...
News
FDA approves Elzonris for blastic plasmacytoid dendritic cell neoplasm
This is the first approved therapy for BPDCN, an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, and can...
Daily News Podcast
Parental leave for residents
Social media can help you stay up-to-date: advice on how to use it.
Conference Coverage
2018: A banner year for hematology drug approvals
SAN DIEGO – There have been 32 new hematology drug approvals and the year isn’t over yet.
News
FDA approves larotrectinib for cancers with NTRK gene fusions
Larotrectinib is approved for patients with solid tumors that have progressed following treatment and harbor a NTRK gene fusion without a...
Video
Technology offers new tools, challenges for rare-disease patients
WASHINGTON –
Video
Education and support enhance care for rare-disease patients
WASHINGTON –
From the Journals
Virus-specific T-cell infusion may resolve progressive multifocal leukoencephalopathy
One patient fully recovered and a second improved but had speech and mobility problems.
Daily News Podcast
Sprain an ankle, get an opioid
And beware a nasty skin souvenir from travel abroad.
Article
Make The Diagnosis - September 2018
A 75-year-old man presented with asymptomatic annular plaques on both arms, thighs, abdomen, and buttocks, present for several months.