Rare Disease

Medicus Pharma advances microneedle doxorubicin therapy to clear multiple Gorlin syndrome BCC lesions.

New IND pushes QRX003 toward first targeted therapy for peeling skin syndrome, with early pediatric results showing rapid healing and no safety issues.

Elizabeth Buzney, MD, discusses the key reasons erythropoietic protoporphyria is frequently misdiagnosed and the consequences of prolonged diagnostic delay.

Elizabeth Buzney, MD, outlines the clinical hallmarks that separate erythropoietic protoporphyria from other photosensitivity disorders, starting with what clinicians won't see on exam.

The SWF Clinical Care Network now includes 27 validated US sites assessed objectively for multidisciplinary capacity across dermatology, neurology, ophthalmology, mental health, and transitional care.

Phase 3 SELVA and phase 2 TOIVA data show Qtorin rapamycin met primary end points for microcystic lymphatic and cutaneous venous malformations.

In this first episode, Elizabeth Buzney, MD, explains why EPP patients suffer for years before receiving an accurate diagnosis, and what clinicians need to know about this debilitating rare disease.

Phase 2 TOIVA data show Palvella's QTORIN rapamycin reduces bleeding and improves quality of life in cutaneous venous malformations, addressing an unmet treatment need.

Dermatology Times' editor in chief highlights essential late-breaker data from AAD 2026, including upadacitinib, rezpegaldesleukin, icotrokinra, zasocitinib, envudeucitinib, sonelokimab, and more.

The case underscores that classic imaging hallmarks of necrotizing fasciitis are frequently absent in early-stage disease, making a high index of clinical suspicion essential for front-line providers.

Even nonadvanced forms of systemic mastocytosis can significantly impact patients’ quality of life, though new treatments are improving outcomes.

Medicus seeks FDA orphan drug status for SkinJect, a dissolvable microneedle doxorubicin patch targeting Gorlin-related basal cell carcinomas, aiming to reduce the need for repeated surgery.

A new case-control study found that patients with pemphigus vulgaris and bullous pemphigoid face more than twice the odds of comorbid acute stress disorder compared with matched controls.

Phase 2/3 brepocitinib trial will target moderate to severe LPP, aiming to calm inflammation and symptoms and potentially deliver the first FDA-approved systemic option.

Kristen Lo Sicco, MD, shares why scarring alopecia can’t wait as LPP demands early treatment to prevent permanent hair loss amid limited FDA-approved options.

At AAD 2026, Lauren Madigan, MD, presented long-term PIONEER trial extension data and urged clinicians to sharpen their recognition of 2 clonally driven inflammatory diseases: indolent systemic mastocytosis and VEXAS syndrome.

LEO Pharma shared 2 posters with data from the ongoing EFFISAYIL ON program, supporting long-term use of spesolimab for generalized pustular psoriasis.

The FDA has granted Priority Review to brepocitinib's NDA for dermatomyositis, with a Q3 2026 PDUFA date that could make it the first drug specifically approved for this indication.

This portion of the AMETHYST trial is the second successful phase 2 study of litifilimab, an investigational monoclonal antibody for CLE.

In a patient with longstanding Hailey-Hailey disease, lebrikizumab was associated with rapid improvement in symptoms and quality of life.

A multicenter trial in China shows ALA-PDT clears Bowen’s disease in 97% with low recurrence, minimal scarring, and high patient satisfaction.

February highlights rare genetic skin diseases, shifting dermatology from supportive care to molecularly targeted therapies.

FDA Fast Track boosts hopes for QRX003 lotion in Netherton Syndrome, with early trials showing rapid skin healing and itch relief.

A combined group of ophthalmology and dermatology specialists traveled to communities in Nepal to conduct consultations, procedures, and teaching sessions, and specifically in dermatology, skin malignancy removals, trainings on biologics and JAKs, and melanoma detection demonstrations.

Priovant Therapeutics' brepocitinib has been assigned a Prescription Drug User Fee Act date in the third quarter of 2026.