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2. Elivaldogene autotemcel - Wikipedia

   en.wikipedia.org/wiki/Elivaldogene_autotemcel

   Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD). It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in May 2018.

3. Adrenoleukodystrophy - Wikipedia

   en.wikipedia.org/wiki/Adrenoleukodystrophy

   For the childhood cerebral form, stem cell transplant and gene therapy are options if the disease is detected early in the clinical course. Adrenal insufficiency in ALD patients can be successfully treated.

4. List of gene therapies - Wikipedia

   en.wikipedia.org/wiki/List_of_gene_therapies

   Elivaldogene autotemcel (Skysona): treatment for cerebral adrenoleukodystrophy [9] Etranacogene dezaparvovec (Hemgenix): AAV-based treatment for hemophilia B [10] Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma

5. Gene therapy - Wikipedia

   en.wikipedia.org/wiki/Gene_therapy

   Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of ... cerebral adrenoleukodystrophy July 2021 exagamglogene ...

6. Leukodystrophy - Wikipedia

   en.wikipedia.org/wiki/Leukodystrophy

   For three types of leukodystrophies (X-linked adrenoleukodystrophy (X-ALD), metachromatic leukodystrophy (MLD) and Krabbe Disease (globoid cell leukodystrophy - GLD), gene therapy using autologous hematopoietic stem cells to transfer the healthy copy of the disease-causing gene with lentiviral vectors has been shown to succeed and has been used ...

7. Why a South Jersey police department stepped in when local 9 ...

   www.aol.com/why-south-jersey-police-department...

   Ayden's life pivoted quickly after a medical emergency on a trip in 2023 led to his April diagnosis of cerebral adrenoleukodystrophy, a rare brain condition for which there is no cure. The only ...

8. bluebird bio - Wikipedia

   en.wikipedia.org/wiki/Bluebird_bio

   bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders. [1]The company's only - in the European Union (EU) - approved drug is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines ...

9. 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 ...