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Neurogene Announces Peer-Reviewed Publication Demonstrating Gene Therapy Candidate NGN-401 with EXACT™ Technology Regulates Transgene Expression in Preclinical Models of Rett Syndrome
02 Apr 2025 20:11 GMT
… therapy to address Rett syndrome and other devastating disorders.”
Rett syndrome is caused by … Overcoming Gene Dosage Sensitivity in Rett Syndrome,” was published in Science … advancement in gene therapy for Rett syndrome, specifically because the disorder …
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Emma’s journey: 110km trek for Rett Syndrome awareness
29 Mar 2025 15:20 GMT
… 110-km Charity Journey Against Rett Syndrome.”
This charitable event involves a … girl named Emma, who has Rett Syndrome, as she travels 110 kilometres … raise awareness about the condition.
Rett Syndrome is a rare genetic condition …
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New Utah laws will help hold child abusers accountable, district attorney says
04 Apr 2025 03:22 GMT
… -old woman who suffered from Rett Syndrome, a neurological condition that caused …
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Why Gov. Cox said these laws are 'crucial' to Utah's public safety
04 Apr 2025 00:42 GMT
… 31-year-old who had Rett Syndrome and for years was a …
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Rett Syndrome Research Trust Appoints Amy Gilliland to its Board of Trustees
13 Mar 2025 11:30 GMT
… to Ashley and all with Rett syndrome.” About Rett Syndrome
Rett Syndrome is a genetic childhood … care. About the Rett Syndrome Research Trust
The Rett Syndrome Research Trust, a … 992d3595-12a0-4c09-8762-7e28bd061583
Rett Syndrome Research Trust Appoints Amy …
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Minoryx doses first patient with leriglitazone in Rett syndrome study
12 Mar 2025 10:44 GMT
… of leriglitazone in treating Rett syndrome. The trial involves 24 … , CEO of Minoryx. “Rett syndrome is one such indication, … the pathways associated with Rett syndrome. Thus far we have … paediatric female patients with Rett syndrome.”
Results from the TREE …
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International Rett Syndrome Foundation Awards Nearly $2 Million in 2024 Grants to Advance Rett Research
11 Mar 2025 13:30 GMT
… visit our website here.
About Rett Syndrome
Rett syndrome is a rare genetic neurological … leading to identifying Rett syndrome's cause, demonstrating Rett syndrome is reversible in …
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Neurogene: Potential To Target Root Cause Of Rett Syndrome With NGN-401
08 Mar 2025 02:54 GMT
… for treatment of patients with Rett syndrome, expected 2nd half of 2025 … with Rett Syndrome, in the 1st half of 2025.
The global Rett Syndrome market …
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Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
05 Mar 2025 14:19 GMT
… of DAYBUE in younger pediatric Rett syndrome patients, including effective strategies to … hand use (57.1%).
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental … al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ …
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Biomed Industries, Inc. Announces Phase 2/3 Results of NA-921, with Superior Efficacy and Safety for Rett Syndrome
04 Mar 2025 11:00 GMT
… improved patient retention rates.
• Rett Syndrome Behavior Questionnaire (RSBQ): The …
AN UNMET NEED IN RETT SYNDROME
Rett syndrome is a severe neuro-developmental … ), and rare diseases, including Rett Syndrome.
For further information, please …