RDR Alert®

With drug breakthroughs reaching epidermolysis bullosa, Paller considers what may come next for other rare skin diseases.

Peter Marinkovich, MD, reflects on the significant growth in advanced therapy development, particularly in rare skin diseases.

Joyce Teng, MD, PhD, discusses how therapeutic advances in fields like epidermolysis bullosa should progress treatment discourse in other rare dermatoses.

Marinkovich discusses how the investigative cell-based graft therapy may serve a key role in the emerging DEB treatment paradigm.

Joyce Teng, MD, PhD, reviews a pair of recently approved agents for DEB—as well as a cell therapy awaiting FDA decision this year.

Gene and cell therapies are reaching patients with the severe forms of epidermolysis bullosa. An expert explains their differing benefits and strengths.

Positive interim data from the TEASE-3 clinical trial show gildeuretinol slows the progression of Stargardt Disease for up to six years.

Califf joins HCPLive to share perspective on AI, gene and cell therapy development, COVID-19, food and substance regulation, maternal and infant health, weight loss intervention, and more.

Phase 3 data presented at ASH 2023 show the gene therapy significantly reduced annualized bleeding rates and the number of total bleeds.

Two experts describe the correlation between the severity of arginine deficiency and worse clinical outcomes among hospitalized children with sickle cell disease experiencing vaso-occlusive pain episodes.

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.

Safer discusses how symptoms and quality of life often go unaddressed in the few clinician-patient interactions about primary sclerosing cholangitis.

Pooled data from The Liver Meeting showed odevixibat sustains itch and serum bile acid benefits in patients with Alagille syndrome. An investigators shares what he wants to learn next.

New pooled phase 3 and extension trial data show odevixibat continues to improve disease management and itch reduction in children with Alagille syndrome.

The UCLA hepatologist discusses the current treatment landscape, prospects for improved diagnostics, and the overlap syndrome live from ACG 2023.

The expanded Indication now includes children of all ages with achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature.

Maralixibat and odevixibat have been "game changers" for their benefit in pruritus, and clinicians are interested to learn more about their long-term hepatic benefit.

Though a regimen of steroids and immunosuppressants are well proven, alternative options may be necessary.

Particularly in children, it's critical that referring physicians keep an awareness for clear symptoms and signs of autoimmune liver disease.

The author of the largest clinical trial for pediatric PFIC to date discusses the various benefit maralixibat provides to disease managementr.

Pooled phase 3 data presented at NASPGHAN 2023 support the benefit-risk profile of the IBAT inhibtor in treating the rare liver disease.

Announced by Ionis Pharmaceuticals, topline results show olezarsen met the study's primary endpoint with a statistically significant reduction in triglyceride levels versus placebo.

Crinetics announced findings from the PATHFNDR-1 trial showing the investigative oral daily drug may provide patients with the rare disease an alternative from recurring injection therapy.

The FDA approval of pozelimab (Veopoz) makes it the first and only treatment indicated for children and adults with CHAPLE disease.