Breakthrough Gene Therapy Saves Infant with Rare Metabolic Disease
May 19th 2025A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ammonia buildup in the blood. The technology used creates an opportunity for the development of personalized treatments for other rare diseases.
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Lack of Social Support Top Barrier to Cell and Gene Therapy Access, Survey Finds
May 12th 2025Healthcare providers are exploring ways to reduce patient travel through a growing trend of expanding sites of administration beyond academic medical centers into community and outpatient settings closer to patients' homes.
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Common Virus Improves Melanoma Treatment Response, Reduces Side Effects
May 9th 2025Patients who were positive for cytomegalovirus had more immune cells circulating in their blood before they started immunotherapy. This may be one reason why these patients responded better to certain immunotherapy treatments, researchers said.
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FDA Approves First-Ever Cell Therapy Treatment for Rare Progressive Eye Disease
March 20th 2025Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells genetically engineered to produce specific therapeutic proteins. It will be available in June 2025.
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Melanoma Treatment Advances Now Crossing into Other Skin Cancers
March 10th 2025Deborah S. Sarnoff, M.D., talks about the recent breakthrough treatments that are paving the way to treat patients with advanced basal cell carcinoma, squamous cell carcinoma and Merkel cell carcinoma, which is a rare and aggressive type of skin cancer.
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Solid Biosciences Reports Positive Data from DMD Gene Therapy Trial
February 19th 2025Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively to muscle cells.
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Blood or Marrow Transplant Recipients Face Higher Skin Cancer Risk, Study Shows
February 13th 2025Risk factors for secondary skin cancer after blood or marrow transplant include those who had treatment with a monoclonal antibody, chronic graft-versus-host disease and posttransplant immunosuppression.
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Social Media Offers Both Promise and Risk in Promoting Skin Cancer Prevention
January 13th 2025In a review of previous studies, researchers found that the images seen on social media contribute to taking actions that are both beneficial with patients seeking dermatological care and unfavorable with increased exposure to UV radiation for a better tan.
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