Rare Diseases
News
FDA gives fast-track approval to new ALS drug
“The FDA’s approval of Qalsody gives me hope that people living with this rare form of ALS could experience a reduction in decline in strength,...
Conference Coverage
Pembrolizumab monotherapy effective for rare melanoma
Patients with unresectable desmoplastic melanoma experienced a response rate that was nearly double other melanomas.
News
Cancer, heart disease vaccines may be ready by 2030, Moderna says
The announcement is yet another sign of what many are calling “the golden age” of vaccine development.
Conference Coverage
Pretransfer visits with pediatric and adult rheumatologists smooth adolescent transition
The time from the last pediatric visit to the first adult visit was substantially shorter for patients who had a dual provider pretransfer visit...
Conference Coverage
Childhood lupus severity linked to social determinants of health
Social determinants of health were linked to lupus disease severity in children, but it will take more than awareness among doctors to address the...
News
CT results in myositis inform cancer screening strategies
Patients with the autoantibody anti–TIF1-gamma had the highest diagnostic yield, while patients under 40 years old had a high frequency of false...
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.
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.