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.

Read more ›

Tags: , , , , , , , ,

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. 

Read more ›

Tags: , , , , , , , , , ,

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,

Read more ›

Tags: , , , , , , , , , , , , , ,

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.,

Read more ›

Tags: , , , , , , , , , ,

Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

Read more ›

Tags: , , , , ,

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.

Read more ›

Tags: , , , , , , , , , , , , , ,

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).

Read more ›

Tags: , , , , , , , , , , ,

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.

Read more ›

Tags: , , , , , , , ,

thasso: conditions

thasso: newest tweets

thasso: recent comments

thasso post: magazine

View my Flipboard Magazine.

thasso: categories

thasso: archives

thasso: simple chat

You must be a registered user to participate in this chat.

  • Targeted therapy pralsetinib achieves high response rates in advanced cancers with RET gene fusions May 29, 2020
    The targeted therapy pralsetinib appears to have high response rates and durable activity in patients with a broad variety of tumors harboring RET gene fusions, according to results from the international Phase I/II ARROW trial, led by researchers at The University of Texas MD Anderson Cancer Center.
  • Genetic data may up diagnostic efficiency for rheumatic disease May 29, 2020
    (HealthDay)—Converting genotype information into an interpretable probability value for five different inflammatory arthritis-causing diseases could potentially improve the diagnostic efficiency of rheumatic disease, according to a study published in the May 27 issue of Science Translational Medicine.
  • New streamlined assay can improve prenatal detection of alpha-thalassemia May 29, 2020
    In a report in The Journal of Molecular Diagnostics, researchers describe a rapid, accurate novel assay for nondeletional alpha-thalassemia genotyping based on one-step nested asymmetric PCR melting curve analysis, which may enhance prenatal diagnosis, newborn screening, and large-scale population screening.
  • Researchers identify mechanisms that make skin a protective barrier May 28, 2020
    A Mount Sinai research team has identified one of the mechanisms that establish the skin as a protective barrier, a breakthrough that is critical to understanding and treating common skin conditions including eczema and psoriasis, according to a study published Thursday, May 28, in the scientific journal Genes & Development.
  • Using electrical stimulus to regulate genes May 28, 2020
    This is how it works. A device containing insulin-producing cells and an electronic control unit is implanted in the body of a diabetic. As soon as the patient eats something and their blood sugar rises, they can use an app on their smartphone to trigger an electrical signal, or they can preconfigure the app do […]
Top