#rare-disease

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#gene-editing #epidermolysis-bullosa #gene-therapy #topical-treatment
Science
fromHarvard Gazette
4 days ago

Family enlists Harvard scientists in fight against brain disease - Harvard Gazette

Gene-editing advances in mice suggest possible future therapies for alternating hemiplegia of childhood while families push research amid limited current understanding and scarce treatments.
Medicine
fromwww.nature.com
3 weeks ago

Gene Therapy Marks a Turning Point for Rare Skin Diseases

Topical gene therapy B-VEC healed chronic wounds and restored mobility and independence for children with epidermolysis bullosa.
Medicine
fromNature
1 month ago

Gene therapy marks a turning point for rare skin diseases

Topical gene therapy B-VEC now enables healing of chronic wounds in epidermolysis bullosa patients, transforming mobility, independence, and paving the way for graft-based treatments.
Women
fromIndependent
2 months ago

'I basically lost my speech language skills. I didn't know how to communicate' - Dublin star Niamh Crowley on recovery from rare disease

Niamh Crowley transitioned from overcoming a rare disease to achieving sports success and academic pursuits.
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