Rare Diseases
Conference Coverage
EULAR systemic sclerosis recommendations now include immunosuppressants
The update to 2017 recommendations include targeted synthetic and biologic DMARDs for the first time.
Latest News
Scientists discover variants, therapy for disabling pansclerotic morphea
The JAK inhibitor ruxolitinib dramatically improved wound healing and rashes in patients with the very rare disorder.
News from the FDA/CDC
FDA approves Yuflyma as ninth adalimumab biosimilar
The manufacturer Celltrion is also seeking an interchangeability designation from the FDA, tentatively expected in the fourth quarter of 2024.
News from the FDA/CDC
FDA approves autoinjector pen for Humira biosimilar, Cyltezo
The drug will be commercially available on July 1, 2023.
Conference Coverage
Noninvasive skin test may aid in Cushing diagnosis
“After proper validation, it could help clinicians in cases which may not be straightforward and could serve as an additional” instrument in the...
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,...
From the Journals
Cocaine damage can be misdiagnosed as nasal vasculitis
In additional to similar nasal symptoms, cocaine can trigger production of antibodies that mimic the clinical presentation of granulomatosis with...
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...