Rare Diseases
Latest News
Phase 3 prurigo nodularis trial shows positive results for nemolizumab
“The use of topical steroids was not allowed in this trial,” Dr. Shawn G. Kwatra said.
News
FDA OKs first drug for Rett syndrome
The drug is designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.
Latest News
Maternal infection in pregnancy ups risk for childhood leukemia?
Maternal infections during pregnancy may be associated with chromosomal and immunologic alterations in the fetus, the authors of the study...
News
VEXAS syndrome: More common, variable, and severe than expected
A large U.S. analysis found that this newly discovered genetic disease has a estimated prevalence of 1 in 4,269 in men older than 50.
News
Mastocytosis: Rare, underdiagnosed, potentially fatal
This often-missed myeloid neoplasm could be deadly and deserves greater attention, experts say.
News
Cancer clinics begin to accommodate patients demanding new cancer detection tests
Sales teams are making a big push for multicancer early detection tests.
News
Have you heard of VEXAS syndrome?
The prevalence of this syndrome is unknown, but it is not so rare.
News
Doctors urge screening for autoimmune disorders for patients with celiac disease
At least in some cases, people with one autoimmune disorder should be tested for others, celiac disease specialists say.
Conference Coverage
A farewell to arms? Drug approvals based on single-arm trials can be flawed
Objective responses, not time-dependent survival outcomes, should be endpoints for single-arm trials, with results only used for conditional...
News
CDC warns of enterovirus strain linked to polio-like condition
The advisory also urged providers to “strongly consider AFM in patients with acute flaccid limb weakness, especially after respiratory illness or...