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#gene-therapy
from24/7 Wall St.
6 days ago
Medicine

Oppenheimer Starts Ocugen (OCGN) at Outperform on Gene Therapy Pipeline

Oppenheimer initiated Outperform coverage of Ocugen with a $10 price target, betting on OCU400's potential 2027 approval as a gene-agnostic treatment for retinitis pigmentosa affecting a broad patient population.
fromNature
6 months ago
Medicine

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.
Medicine
from24/7 Wall St.
6 days ago

Oppenheimer Starts Ocugen (OCGN) at Outperform on Gene Therapy Pipeline

Oppenheimer initiated Outperform coverage of Ocugen with a $10 price target, betting on OCU400's potential 2027 approval as a gene-agnostic treatment for retinitis pigmentosa affecting a broad patient population.
more#gene-therapy
fromwww.bbc.com
3 months ago

Christmas uncertainty for family of girl with rare condition

"The Gosh charity gave Zarwa a really amazing Christmas," Fahima said. "She went to sleep and woke up to find her ward covered in twinkling lights and decorations. She had a stocking packed with gifts."
UK news
Science
fromHarvard Gazette
5 months 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
6 months 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.
Women
fromIndependent
8 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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