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CRISPR-Based Gene Therapy Earns Beta Thalassemia Approval

Exa-cel earned its second FDA approval. The one-time therapy, which has a list price of $2.2 million in the United States, should be available for patients early this year. ....

United States , Drug Administration , Vertex Pharmaceutical , Genomics Genomic Medicine , Beta Thalassemia , Clustered Regularly Interspaced Short Palindromic Repeats , Crispr Cas9 , Thalassemia Thalassaemia , Biologic Therapy , Gene Therapy , Transplantation Of Stem Cells , Ransplantation Of Hematopoietic Stem Cells , Hematopoietic Stem Cell Transplant , Stem Cell Transplantation , Stem Cell Therapy ,

EU Conditional Approval: Gene Editor to Treat Blood Diseases

The single-dose therapy involves treating and editing patient stem cells ex vivo, then reinfusing them. ....

Vertex Pharmaceuticals Ireland , European Medicines Agency , Drug Administration , More Research , Sickle Cell Disease , Ickle Cell Anemia Sickle Anaemia , Clustered Regularly Interspaced Short Palindromic Repeats , Crispr Cas9 , Gene Editing , Genetic Editing , Rare Diseases , Transplantation Of Stem Cells , Ransplantation Of Hematopoietic Stem Cells , Hematopoietic Stem Cell Transplant , Stem Cell Transplantation , Stem Cell Therapy , Tem Cell Haematopoietic Stem Transplant ,

Sickle Cell CRISPR Therapy May Offer 'Functional Cure'

Patients with severe sickle cell disease could soon be freed from vaso-occlusive crises after receiving an infusion of exa-cel, the first gene-editing therapy to use CRISPR technology. ....

United States , Sarahh Obrien , Haydar Frangoul , Akshay Sharma , Vertex Pharmaceuticals , American Society Of Hematology , Drug Administration , Jude Children Research Hospital In Memphis , Tristar Centennial , American Society , Nationwide Children , Jude Children , Research Hospital , Genomics Genomic Medicine , Sickle Cell Disease , Ickle Cell Anemia Sickle Anaemia , Clustered Regularly Interspaced Short Palindromic Repeats , Crispr Cas9 , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii , Ol Ii , Lysosomal Storage Disease , Emoglobin Hgb , B Hemoglobin ,

Genetic Therapies Bring Change to Neurology Clinics

New therapies are on the horizon for genetic neuromuscular diseases, and this will raise both hopes for patients and challenges for neurologists. ....

United States , Nicolas Madigan , American Association For Neuromuscular , American Association , Electrodiagnostic Medicine , Mayo Clinic , Genomic Medicine , Edicine Genomic , Gene Editing , Genetic Editing , Genetic Testing , Muscular Atrophy , Uscular Atrophies , Chronic Inflammatory Demyelinating Polyradiculoneuropathy , Chronic Inflammatory Demyelinating Polyneuropathy , Clustered Regularly Interspaced Short Palindromic Repeats , Crispr Cas9 , Clinical Research , Clinical Trials , Linical Studies ,

More Evidence Metformin May Be Neuroprotective

Stopping metformin was linked to an increased risk for dementia diagnosis in a large cohort of adults with T2D, providing more evidence of the drug’s potential neuroprotective effects. ....

Scott Zimmerman , Gilead Sciences , National Institutes Of Health , Abbott Laboratories , Abbvie Inc , University Of California San Francisco , National Institute On , Permanente Northern California , California San Francisco , National Institutes , National Institute , Alzheimers Disease , Lzheimer Disease , Diabetes Mellitus Type 2 , Iabetes Mellitus Type Ii , Type 2 Diabetes , Elderly Concerns Of Older Adults , Genomic Medicine , Edicine Genomic , Clustered Regularly Interspaced Short Palindromic Repeats , Crispr Cas9 , Geriatrics Elder Care , Medical Records ,