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  2. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma; Idecabtagene vicleucel (Abecma): treatment for multiple myeloma [12] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [11] Nadofaragene firadenovec (Adstiladrin): treatment for ...

  3. Gene therapy - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy

    Gene therapy approaches to replace a faulty gene with a healthy gene have been proposed and are being studied for treating some genetic diseases. As of 2017, 11.1% of gene therapy clinical trials targeted monogenic diseases.

  4. List of genetic disorders - Wikipedia

    en.wikipedia.org/wiki/List_of_genetic_disorders

    The following is a list of genetic disorders and if known, type of mutation and for the chromosome involved. Although the parlance "disease-causing gene" is common, it is the occurrence of an abnormality in the parents that causes the impairment to develop within the child. There are over 6,000 known genetic disorders in humans.

  5. FDA approves Pfizer's first gene therapy for rare inherited ...

    www.aol.com/news/fda-approves-pfizer-first-gene...

    The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...

  6. US FDA approves Vertex/CRISPR gene therapy for an inherited ...

    www.aol.com/news/us-fda-approves-vertex-crispr...

    The decision earns the therapy, branded as Casgevy, the second U.S. approval after it was greenlighted in December for sickle cell disease, another inherited blood disorder.

  7. Gene therapy may cure rare diseases. But drugmakers ... - AOL

    www.aol.com/news/gene-therapy-may-cure-rare...

    Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. In 2022, London-based Orchard Therapeutics stopped investing in an ...

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