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Gene Therapy Offers Hope for Australian Children with Epidermolysis Bullosa
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Two days ago the FDA approved the first gene therapy for people ages 2 & older for my disease, Spinal Muscular Atrophy, Itvisma. This is huge. Groundbreaking. Life changing. This gene therapy was approved in a slightly different form for babies born with SMA 2 & younger in 2019. I’ve been digesting this news for the past two days, reading more information & thoughts from other adults with SMA. I have previously been on Spinraza and I am currently taking a Evrysdi (a daily oral medication). Both
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Vyjuvek is the first FDA-approved topical gene therapy and the first treatment for DEB (approved in 2023). It uses a modified/stripped version of the HSV virus to carry the instructions for collagen VII to the cells of the wound bed so that they can produce the collagen as the skin heals. I first used in 2023/2024 after a hand surgery and found it really helpful for healing my hand but not as helpful for other chronic/recurrent wounds. I decided to restart it again after my recent surgery on my
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