Rare Disease

A case report presents a unique case of Bowen's disease affecting the lower extremities in a 75-year-old woman, which was initially misdiagnosed as a fungal infection.

Karen McGuire, PhD, discusses TAMES-02, a clinical trial evaluating TolaSure for Epidermolysis Bullosa Simplex.

Orphan designation provides Soligenix with potential regulatory, financial, and exclusivity benefits as it develops SGX945.

Shannon C. Trotter, DO, FAOCD, FAAD, and patient Janene Tirado, highlight GPP Awareness Day and the urgent need for recognition and understanding of generalized pustular psoriasis.

BioMendics initiates TAMES-02 trial for TolaSure, a groundbreaking therapy targeting the root cause of Epidermolysis Bullosa Simplex, offering new hope.

The open-label trial showed dusquetide produced similar results to apremilast in reducing oral ulcers, pain, and ulcer duration.

PC111, a novel therapy from Scinai, targets pemphigus vulgaris and SJS/TEN, offering rapid relief without immunosuppression.

Quoin Pharmaceuticals advances QRX003 for Netherton Syndrome, receiving FDA's RPD Designation and showing promising clinical results.

Galderma launches clinical trials for nemolizumab, targeting systemic sclerosis and chronic pruritus of unknown origin, addressing critical patient needs.

Abeona Therapeutics' VIITAL trial reveals promising results for RDEB treatment, showcasing significant wound healing and pain relief with Zevaskyn therapy.

A new partnership aims to revolutionize topical treatments for epidermolysis bullosa, enhancing patient care with innovative drug delivery technology.

Quoin Pharmaceuticals advances QRX003 for Netherton Syndrome, receiving FDA clearance for a pivotal trial to assess its effectiveness and safety.

ZEVASKYN, the first FDA-approved gene therapy for wounds in recessive dystrophic epidermolysis bullosa, is now available at Lurie Children's Hospital.

A pediatric patient with Peeling Skin Syndrome observed overall skin healing with no adverse events after 12 weeks of treatment.

The FDA approved pz-cel, a groundbreaking gene therapy for recessive dystrophic epidermolysis bullosa, transforming treatment options for patients.

The pilot study showed notable decreases in mean melanin index after 3 sessions of PRP injections.

The presentation, led by Amy Paller, MS, MD, highlighted the upcoming phase 3 SELVA study for microcystic lymphatic malformations.

A new case report demonstrated the JAK inhibitor’s efficacy at improving disease severity up to 6 months.

The study stated early recognition and aggressive treatment of childhood-onset MMP are essential to prevent permanent scarring.

The novel treatments will be for patients with orphan conditions like Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome, and palmoplantar keratoderma.

Nanoneedles boosted CRISPR base editing efficiency in RDEB fibroblasts, enabling precise COL7A1 correction and improved cell function.

Efficacy, safety, and pharmacokinetic data support the use of vilobelimab in treating pyoderma gangrenosum and hidradenitis suppurativa.

The novel formulations will harness Quoin’s Invisicare technology to treat microcystic lymphatic malformations, venous malformations, and angiofibromas.