Rare Diseases
News
FDA OKs new drug for Fabry disease
“The totality of clinical data suggests that Elfabrio has the potential to be a long-lasting therapy.”
From the Journals
Cutaneous vasculitis curtails quality of life
Survey respondents described a generally poor health-related quality of life that was detailed as more severe with a skin-specific instrument than...
From the Journals
Study shifts burden of IgG4-related disease to women
First population-based U.S. analysis establishes “key benchmarks” for immune-mediated condition initially described in 2001.
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,...
Latest News
Study compares noninvasive treatments of cutaneous neurofibromas
The epidermis overlying cNF is normal, “so there is no reason to use nonselective or surgical methods and destroy a perfectly good epidermis when...
Conference Coverage
Answers sought for mental health challenges in pediatric rheumatology patients
One study found a tremendous increase in screening by automating the process while another found remote delivery of a 6-week cognitive-behavioral...
Latest 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
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...
Feature
Biosimilars and patients: Discussions should address safety, cost, and anxiety about change
Physicians can respond to pushback from patients on switching to a biosimilar by offering resources and discussing options for both biosimilar and...
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.