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Vertex Pharma (VRTX) Reports Positive CHMP Opinion for CASGEVY

Vertex Pharma (VRTX) Reports Positive CHMP Opinion for CASGEVY
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Rome , Lazio , Italy , Franco-locatelli , European-medicines-agency , European-commission , European-union , Vertex-pharmaceuticals-incorporated-nasdaq , Catholic-university-of-the-sacred-heart , Department-of-pediatric-hematology , Committee-for-medicinal-products-human-use , Vertex-pharmaceuticals-incorporated

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 , American , 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

Dr Franco Locatelli Discusses the Treatment of Patients with cGvHD

Franco Locatelli, MD, PhD, head of the Department of Pediatric Hematology and Oncology at IRCCS Bambino Gesu Children’s Hospital in Rome, discusses the treatment of both adult and pediatric patients with chronic graft versus host disease (cGvHD).

Rome , Lazio , Italy , Franco-locatelli , Department-of-pediatric-hematology , University-of-rome , Pediatric-hematology , Gesu-children , Rare-disease , Cgvhd , Hematology , European-hematology-association

Dr Franco Locatelli: Assessing Ruxolitinib Treatment in Pediatric Patients With cGvHD

Franco Locatelli, MD, PhD, a professor and hematologist from Italy, discusses results from the the phase 2 REACH 5 studies assessing the use of ruxolitinib in pediatric patients with chronic graft versus host disease (cGvHD).

Italy , Rome , Lazio , Franco-locatelli , University-of-rome , Department-of-pediatric-hematology , Pediatric-hematology , Gesu-children , Rare-disease , Cgvhd , Chronic-graft-versus-host-disease , Ruxolitinib

Exa-Cel Therapy 'Transformative' in SCD, Beta-Thalassemia

The investigational gene therapy exagamglogene autotemcel shows encouraging efficacy, safety, and patient-reported outcomes for patients with transfusion-dependent beta-thalassemia and sickle cell disease.

Rome , Lazio , Italy , Haydar-frangoul , Franco-locatelli , Raffaella-colombatti , Michaelj-eckrich , Sarah-cannon-center , European-hematology-association , Atrium-health-levine-children-hospital-cancer , University-of-padova , Catholic-university-of-the-sacred-heart

FDA Set to Review Exagamglogene Autotemcel for Sickle Cell Disease, Transfusion-Dependent Beta Thalassemia

The FDA has granted priority and standard review designations to the biologics license applications for exagamglogene autotemcel for the treatment of patients with sickle cell disease and transfusion-dependent beta thalassemia, respectively, marking the first CRISPR gene editing filings to be accepted for review by the FDA.

Carmen-bozic , Franco-locatelli , Vertex-pharmaceuticals , Global-medicines-development , Sapienza-university-of-rome , Department-of-pediatric-hematology , Healthcare-products-regulatory-agency , European-hematology-association , European-medicines-agency , Medical-affairs , Regenerative-medicine-advanced-therapy , Rare-pediatric-disease

Vertex Pharmaceuticals Incorporated: Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress

Both trials met the primary and key secondary endpoints at the pre-specified interim analysis - Data continue to demonstrate transformative and durable benefit - Safety profile consistent with busulfan

Australia , London , City-of , United-kingdom , Cambridge , Cambridgeshire , San-francisco , California , United-states , Boston , Massachusetts , Zeiten

Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress

- Both trials met the primary and key secondary endpoints at the pre-specified interim analysis -

- Data continue to demonstrate transformative and durable benefit -

- Safety profile consistent... | June 9, 2023

Massachusetts , United-states , Boston , California , Switzerland , Germany , Italy , London , City-of , United-kingdom , Australia , San-francisco

The 21st Century may see the death of cancer. (But it might come sooner under a more rational system.) – The Greanville Post

The 21st Century may see the death of cancer. (But it might come sooner under a more rational system.) – The Greanville Post
greanvillepost.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from greanvillepost.com Daily Mail and Mail on Sunday newspapers.

Rome , Lazio , Italy , Pennsylvania , United-states , Italian , Robbie-majzner , Franco-locatelli , Bambino-gesu , Stanford-university-school-of-medicine , Drug-administration , University-of-pennsylvania-dr

Novel treatment shows promise against rare cancer in kids

A novel treatment using supercharged immune cells appears to work against tumors in children with a rare kind of cancer.

Researchers reported the finding Wednesday in the New England Journal of Medicine. Nine of 27 children in the Italian study had no sign of cancer six weeks after treatment, although two later relapsed and died. The treatment is called CAR-T cell therapy. It's already used to fight leukemia and other cancers in the blood. Experts say this study in children with neuroblastoma is the first time the therapy has worked well in solid tumors. That raises hopes that it can be used against other kinds of cancer.

Pennsylvania , United-states , Rome , Lazio , Italy , Italian , Franco-locatelli , Robbie-majzner , Bambino-gesu , University-of-pennsylvania-dr , Drug-administration , Stanford-university-school-of-medicine