#rare-disease

#rare-disease

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.

Topical gene therapy B-VEC healed chronic wounds and restored mobility and independence for children with epidermolysis bullosa.

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.

Progeria could potentially be cured through new gene-editing treatments.

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