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Sickle Cell Gene Therapy 'Truly Transformative'

Patients who received lovo-cel had durable remissions and reductions in vaso-occlusive events, pain crises, and hospitalizations in the longest running gene therapy trial for sickle cell disease. ....

United States , Ann Arbor , Sarah Obrien , Julie Kanter , University Of Michigan School Medicine , University Of Alabama , American Society Of Hematology , Vertex Pharmaceuticals , Drug Administration , American Society , Saraho Brien , Nationwide Children , Crispr Therapeutics , Steven Pipe , Michigan School , Genomics Genomic Medicine , Sickle Cell Disease , Ickle Cell Anemia Sickle Anaemia , Gene Therapy , Biologic Therapy , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii , Ol Ii , Lysosomal Storage Disease ,

Patients With hEDS Report Skin Laxity, Scarring

Follicular occlusion, mast cell disease, and eczema were common presentations among patients with hypermobile Ehlers Danlos syndrome. ....

United States , Alan Snyder , Ehlers Danlos , Journal Of The American Academy Dermatology , University Of South Carolina , Medical University , South Carolina , American Academy , Urticaria And Angioedema Angio Oedema , Skin And Soft Tissue Infection , Wound Management , Wound Care , Wound Healing , Wound Repair , Dermatologic Surgery Dermatological Cutaneous Dermasurgery , Ehlers Danlos Syndrome , Acne Vulgaris , Acneiform Eruptions , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii ,

FDA OKs Two Gene-Editing Therapies for Sickle Cell Disease

The approval for the two gene-editing treatments, called exa-cel and lovo-cel, could help thousands with the blood disorder. ....

United States , Roberta Brodsky , Franco Locatelli , Nicole Verdun , Johns Hopkins University School Of Medicine , Sapienza University Of Rome , Vertex Pharmaceuticals , Drug Administration , American Society Of Hematology , Crispr Therapeutics , Johns Hopkins University School , American Society , Biologics Evaluation , Sapienza University , Genomics Genomic Medicine , Sickle Cell Disease , Ickle Cell Anemia Sickle Anaemia , Lysosomal Storage Disease , Blood Transfusion , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii , Ol Ii , Transplantation Of Stem Cells , Ransplantation Of Hematopoietic Stem Cells ,

Children With Sickle Cell Disease at Risk for Vision Loss

Previously thought to be age-dependent, retinopathy affects a full third of children with sickle cell disease, a new study found. ....

United States , Mary Ellen Hoehn , American Academy Of Ophthalmology , University Of Tennessee Health Science Center , American Academy , Ellen Hoehn , Tennessee Health Science Center , Sickle Cell , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii , Ol Ii , Lysosomal Storage Disease , Retinal Disease , Vision Care And Maintenance , Phthalmic Route Of Administration , Phthalmic Delivery , Retinal Artery Occlusion , Preventive Screening , Retinal Detachment ,

Rethinking Approach to Manage Fever in ED for Children With Sickle Cell Disease

Lower risk of bacteremia in children with sickle cell disease (SCD) presenting to the emergency department (ED) with fever calls critical reexamination of long standing approach towards management of SCD in children. ....

Johnj Strouse , Department Of Medicine , Duke University , Division Of Hematology , Stephen Rinner , Bloodstream Infections , Febrile Child , Hild With Fever , Pediatric Fever , Bacteremia Bacteraemia , Erectile Dysfunction , Rectile Dysfunction Ed , Ebrile Response , Sickle Cell Disease , Ickle Cell Anemia Sickle Anaemia , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii , Ol Ii ,