Blog Archives

Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data?

August 13, 2019 – Onasemnogene abeparvovec-xioi (Zolgensma) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Gene therapy: Zolgensma to treat spinal muscular atrophy

May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at or before birth while others are not apparent until adulthood. 

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Marsili Syndrome: A mutation in the ZFHX2 gene leaves Italian family painless

December 21, 2017 – No pain on gain. This headline might not at all apply for this Italian family. In a paper just published in the Journal Brain,

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Voretigene Neparvovec-Rzyl (Luxturna): Gene therapy to treat inherited vision loss

December 20, 2017 –  The American Food & Drug Administration (FDA) has just approved  a novel gene therapy to treat patients with a rare form of inherited vision loss. Voretigene Neparvovec-Rzyl (Luxturna) is the first directly administered gene therapy approved to target a disease caused by mutations in a specific human gene (i.e.,

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Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

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Axicabtagene Ciloleucel (Yescarta) gene therapy for diffuse large B-cell lymphoma

October 19, 2017 – The American  Food & Drug Administration (FDA) just approved Axicabtagene Ciloleucel (Yescarta), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment.

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The first gene therapy FDA approved in the US

September 3, 2017 – For a very first time, the American Food & Drug Administration (FDA) has approved a gene therapy for the United States (US) in that it approved CAR T-cell therapy to treat certain children and young adults with B-cell acute lymphoblastic leukemia (B-cell ALL).

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New hope for children with the ultra-rare immune disorder ADA-SCID: Gene therapy Strimvelis recommended for approval by EMA’s CHMP

April 02, 2016 – The European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union (EU) for a new gene therapy for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant.

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