Rare Diseases
Latest News
High early recurrence rates with Merkel cell carcinoma
Approximately 95% of all recurrences happened within 3 years of the initial diagnosis in the prospective cohort study.
Latest News
U.S. FDA declines to approve Amryt’s topical gel for rare skin diseases
The decision comes after the FDA had extended its review of the treatment, oleogel-S10, in November by three months to review additional analyses...
Feature
Cystic fibrosis in retreat, but still unbeaten
Latest News
Lipedema: A potentially devastating, often unrecognized disease
“If patients with lipedema gain weight, the problem becomes even worse,” Dr. C. William Hanke said.
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Superficial radiotherapy tied to remission, better quality of life in severe Hailey-Hailey disease
To explore the use of superficial radiotherapy, the investigators analyzed data on 13 patients who received the treatment.
News
FDA approves levoketoconazole for Cushing syndrome
The drug, which inhibits cortisol synthesis, is seen as addressing an unmet need.
From the Journals
Case series show no consensus on treatment for palmoplantar pustulosis, generalized pustular psoriasis
“These case series confirm the rarity of both generalized pustular psoriasis and palmoplantar pustulosis ... and the lack of effective treatment...
Latest News
Triple therapy provides robust and durable benefits in cystic fibrosis
From baseline through 24 weeks, the mean Cystic Fibrosis Questionnaire–Revised (CFQ-R) respiratory domain score - the primary endpoint - increased...
From the Journals
Spesolimab speeds lesion clearance in generalized pustular psoriasis
Patients treated with intravenous spesolimab showed significant clearance, but also susceptibility to infections.
From the Journals
Prurigo nodularis has two disease endotypes, a cluster analysis shows
Black patients with PN appear more likely to have a distinct systemic inflammatory signature.
Latest News
New option for flares in pustular psoriasis
Spesolimab earns Priority Review status from the FDA for treatment of flares in generalized pustular psoriasis.