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 ...
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 ...
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.
Neurogene hits a setback in the clinic, one week after raising funds
The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side ...
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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 ...
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 ...
Neurogene Shares Drop 35% on Rett Syndrome Gene Therapy Trial Data
Neurogene ( NGNE, Financials) shares fell 35% in Tuesday's pre-market trading following the release of interim results from a ...
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Neurogene’s (NGNE) Surprising Setback: What’s Behind the Stock Drop?
When looking at Neurogene’s stock performance year-to-date, it’s hard to ignore the impressive +269.09% increase. However, this recent downturn suggests that the market is responding to something ...
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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 ...
Falkirk Herald on MSN13h
Stunning Wallace Monument photograph wins national award for Falkirk district's Kilted Photographer
A stunning image of the Wallace Monument at sunset has won a Redding photographer a national award. Thomas Lamont – known ...
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Throckley mother's fight for Batten Disease drug to save 'beacon of hope' daughter
Gail Rich's daughters Jessica and Nicole were both diagnosed with a rare and incurable neurodegenerative condition, commonly known as Batten Disease. The family's battle for funding and access to ...
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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, ...