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Tag: Gene Therapy

Breyanzi Approved for Certain Types of Large B-Cell Lymphoma

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The CAR T cell therapy OK'd for patients who have not responded to or have relapsed after receiving two or more other systemic treatments

A breakthrough gene therapy will be covered for certain types of lymphoma and leukemia

Medicare to Cover CAR-T Therapy for Leukemia, Lymphoma

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Chimeric antigen receptor T-cell therapy boosts a patient's own immune cells
The maker of Zolgensma (onasemnogene abeparvovec-xioi) gave manipulated data to the U.S. Food and Drug Administration when it approved the drug

FDA: Approval of Zolgensma Was Based on Manipulated Data

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Despite inaccurate data, FDA says it is 'confident that Zolgensma should remain on the market'
Its extremely high price tag means that a lifesaving medication to treat young children with spinal muscular atrophy is simply too expensive for most families.

Lifesaving Drug for Infants Costs $2.1 Million a Dose

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When Zolgensma was approved, maker Novartis said it expected insurance companies to cover the cost
The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy

FDA Approves First Gene Therapy for Spinal Muscular Atrophy

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Patients treated with Zolgensma have shown improvement in reaching developmental motor milestones
For patients with Parkinson's disease undergoing gene therapy

Gene Therapy Forms New Polysynaptic Pathways in Parkinson’s

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Patients receiving GAD gene therapy develop unique treatment-dependent polysynaptic brain circuit
The potential first gene therapy in the United States is being reviewed by a U.S. Food and Drug Administration expert panel.

Potential First U.S. Gene Therapy Now Under FDA Review

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Tisagenlecleucel could be a one-time treatment for children and young adults with advanced leukemia
Gene therapy may benefit children and teens with Wiskott-Aldrich syndrome

Gene Tx May Benefit Children, Teens With Wiskott-Aldrich Sx

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Patient's own blood stem cells are removed to correct the WAS gene, then injected back into the patient