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Elna Saah, MD: Unraveling the Current Landscape in Sickle Cell Disease
18 Apr 2024 22:59 GMT
… day, from early trial data indicating the proficiency … US Food and Drug Administration (FDA) approvals, including … /facts.html.
Sickle cell disease. Hematology.org. Accessed April … /anemia/sickle-cell-disease.
Treatment. National Heart Lung and …
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Innovative Gene Editing Approach to Sickle Cell Disease Wins National Clinical Research Award
18 Apr 2024 20:12 GMT
… develop the disease. The average lifespan of an individual with sickle-cell disease … HBG2 Promoters to Treat Sickle Cell Disease, used an … , a proven treatment for sickle-cell disease. Gene-edited … center randomized clinical trial, which included more …
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Boy with rare blood disorder receives new life-changing gene therapy treatment
19 Apr 2024 02:05 GMT
… undergo gene therapy treatment for a rare … ;Historically, the only treatments were medical management with … and transformative gene therapy treatment. Dr. Banu Aygun … treatment to prevent, treat or cure certain hereditary diseases, including sickle-cell anemia …
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Novel cell therapy treatments offer promise to immune-compromised children
18 Apr 2024 14:30 GMT
… Treatment Consortium (PIDTC) to create the first multi-center, pediatric-consortium trial … as sickle cell disease. While doctors can treat some immune … the way for commercial development.” The Good Manufacturing … the Food and Drug Administration to identify …
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Does This Acquisition Make Vertex Pharmaceuticals Stock a Buy?
18 Apr 2024 12:47 GMT
… pipeline.
Biotech giant Vertex Pharmaceuticals (VRTX -0.27% … a drug?
Alpine Immune Sciences focuses on developing … its leading candidate in treating IgAN. In the … sickle cell disease and beta-thalassemia, two rare blood disorders. This gene-editing treatment …
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New sickle cell treatment center opens at BJC
18 Apr 2024 03:09 GMT
… the 12th sickle cell treatment center in the country. … medication required.
“It is very important for patients with sickle cell disease … who are specialized to treat this disease,” Dr. … care of patients with sickle cell disease.”
Those who are looking …
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Akshay Sharma, MBBS, on the Rapidly Expanding Lanscape of Care for Sickle Cell Disease
15 Apr 2024 14:17 GMT
… only drug on the market for patients with sickle cell disease ( … December 2023: Vertex Pharmaceuticals'; and CRISPR Therapeutics … approaches to treating SCD are now in clinical development, as … another potentially curative treatment option for SCD, may …
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Long Island boy with rare blood disorder undergoes gene therapy
18 Apr 2024 23:39 GMT
… — Until now, the best treatment for some diseases has been … patient’s cells for treatment.
Dr. Banu Aygun, … successfully to treat many other diseases, including Sickle Cell Anemia. Ten … years in the making, this new treatment marks …
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Skin in the Game: Oruka Takes on Blockbuster Drugs for Psoriasis, Dermatological Disorders
15 Apr 2024 21:09 GMT
… focused on treating plaque … (ustekinumab), and Sun Pharmaceutical Industries’ Ilumya® … FDA’s first-ever approval for a CRISPR-edited therapy in sickle cell disease … treatments for inflammatory bowel disease, and Apogee a developer of biologics to treat …
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Genovefa (Zenia) Papanicolaou, MD, on Managing Infectious Diseases in Patients Receiving Stem Cell Transplant or Cell Therapy
18 Apr 2024 17:01 GMT
… nonmalignant hematological diseases like sickle cell disease. Although these treatment options can have a … system, especially during the immediate treatment period. As such, patients receiving … results from a phase 1 trial of multivirus-specific T-cells …