Rare Diseases
News
FDA OKs first-ever topical gene therapy, for rare skin disease
“Because it’s safe and easy to apply directly to wounds, it doesn’t require a lot of supporting technology or specialized expertise, making...
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.”
News
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...
News
IVIG shows no impact on VTE risk in dermatomyositis patients
The rate of venous thromboembolism did not increase in dermatomyositis patients treated with intravenous immunoglobulin, compared with those not...
Conference Coverage
Low disease state for childhood lupus approaches validation
A new childhood version of the Lupus Low Disease Activity State has been purposefully developed to align with the criteria already used for adults...
News
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
New outbreaks of Marburg virus disease: What clinicians need to know
Overall, the risk of MVD in nonendemic countries, like the United States, is low but there is still a risk of importation.
Conference Coverage
U.S. incidence, prevalence of myasthenia gravis is rising
These new estimates are “significantly higher” than those from a prior U.S. analysis from 2003.
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,...