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Throughout 2023, interest in the application of CAR-T and other cell therapies to autoimmune disease skyrocketed, with a multitude of clinical trials being initiated by various companies.

The Lupus Approach: CAR-T Begins the Jump from Blood Cancers to Autoimmune Disease

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.

Reviewing 2023 with FDA Commissioner Robert M. Califf, MD

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

Etranacogene Dezaparvovec Significantly Improves Hemophilia B Past 2 Years

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.







Low Arginine Bioavailability Predicts Poor Clinical Outcomes in Sickle Cell Disease

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.

After Elevidys: DMD Advocacy Past First Gene Therapy Approval

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

FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors

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

Ricky Safer: What Clinicians Need to Know About PSC

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.

Ryan T. Fischer, MD: Long-Term Odevixibat Benefit for Alagille Syndrome

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

Odevixibat Maintains Alagille Syndrome Pruritus, Bile Acid Reductions at 36 Weeks

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

Autoimmune Hepatitis at ACG 2023, with Gina Choi, MD

Vasculitis

Amyloidosis

The HCPLive amyloidosis page is a resource for medical news and expert insights on the rare disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for amyloid buildup, and more.

Huntington Disease

On the HCPLive Huntington disease page, resources on the topics of medical news and expert insight into HD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on Huntington's chorea, and more.

Lysosomal Storage Disorders

Lysosomal storage disorders page, resources on the topics of medical news and expert insight into the rare diseases can be found. Content includes articles, interviews, videos, podcasts, and breaking news on lysosomal disorder disease research, treatment, and drug development.

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

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