Treatment to preserve sight in children with rare genetic disorder shows promise in clinical trial
A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...
pharmaphorum10h
Neurogene shares fall on Rett syndrome gene therapy data
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but ...
Armed robbery in Revesby12h
New Hope to Prevent Blindness in Kids With Rare Disease
A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialled by clinicians and researchers at UCL and Great Ormond Street Hospital (GOSH). The study, ...
Neurogene Stock Tanks After Adverse Event In Early-Stage Rett Syndrome Gene Therapy Trial
Neurogene reports interim Phase 1/2 trial results for NGN-401 gene therapy in treating pediatric Rett syndrome, showing ...
clinicaltrialsarena15h
Neurogene’s stock sinks after adverse event with Rett syndrome gene therapy
Neurogene's stock price collapsed by 34.8% in pre-market trading after announcing data from the Phase I/II Rett syndrome ...
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Neurogene Dropping Batten Disease Gene Therapy; Discloses Adverse Event in Rett Syndrome Trial
NEW YORK – Neurogene on Monday said after the market closed that it will stop advancing NGN-101 as a treatment for CLN5 Batten disease, a very rare genetic disorder. The New York-based company said it ...
BioPharma Dive1d
Neurogene hits a setback in the clinic, one week after raising funds
Separately, Neurogene also shared its plans to discontinue development of a gene therapy for Batten disease after the Food ...
Hunts Post1d
St Ives shop raises £300 for Caitlin Passey with donations
The Refill Shop of Ikigai collected the money for Safe Haven 4 Caitlin through loyalty card donations over six months. Caitlin Passey, aged 10, was diagnosed with CLN3 Batten disease in 2021. This ...
Neurogene: Interesting CNS Disease Company, Data Coming Soon
Neurogene has a $903M market cap with two phase 1/2 assets, NGN-401 for Rett Syndrome and NGN-101 for CLN5 Batten Disease.
Developing tailor-made treatments for ultra-rare disease patients: Research provides a roadmap
In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...
Nature9d
Neurodegenerative diseases articles from across Nature Portfolio
Neurodegenerative diseases are a heterogeneous group of disorders that are characterized by the progressive degeneration of the structure and function of the central nervous system or peripheral ...
Variety14d
Lady Gaga Fights Different Versions of Herself in New Video for ‘Disease’
Shortly after putting out her dark electro new single “Disease,” Lady Gaga has shared its official music video. In the newly-released visuals, Gaga confronts different versions of herself ...