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WebREGENXBIO's Hunter Syndrome Gene Therapy Hit With New FDA Clinical Hold Over Spine MRI Findings
9+ hour, 42+ min ago (605+ words) BioBuzz REGENXBIO’s Hunter Syndrome Gene Therapy Hit With New FDA Clinical Hold Over Spine MRI Findings REGENXBIO, the Rockville, Maryland-based gene therapy company, announced that the FDA has placed a clinical hold on RGX-121, its investigational one-time gene therapy for…...
EC approves Acadia's Daybu as first-ever Rett syndrome treatment in the EU
15+ hour, 4+ min ago (376+ words) AllSci The European Commission has granted San Diego-based Acadia Pharmaceuticals (Nasdaq: ACAD) marketing authorization for Daybu (trofinetide) as a treatment for the rare genetic neurodevelopmental disorder Rett syndrome. Daybu becomes the first drug approved for Rett syndrome in the EU....
Pathogenic MECP2 Variants Reveal Broader Neurological Spectrum Beyond Rett
7+ hour, 20+ min ago (827+ words) Syndrome Bioengineer.org...
Measuring Muscles with Quantitative MRI: New Study Published in Neurology Expands the Evidence for Using MRI Biomarkers in SBMA
17+ hour, 5+ min ago (210+ words) Eagle-Tribune State Rep. Paulino arrested for COVID-19 fraud following Lawrence mayor AMRA Medical - Measuring Muscles with Quantitative MRI LINKÃPING, Sweden--(BUSINESS WIRE)--Aug 26, 2026-- AMRA Medical is pleased to announce the publication of new research in Neurology demonstrating the potential…...
How rare genetic variants could uncover new drug targets
22+ hour, 32+ min ago (1241+ words) Most common traits are influenced by many genetic variants with small effects. A new study suggests that unusually high or low levels of traits such as cholesterol or blood pressure may instead be driven by rare genetic variants with much…...
Bupa to give genetic tests to prevent serious diseases - AOL
1+ day, 1+ hour ago (691+ words) Britain’s biggest private medical insurer is to start offering genetic testing and personalised prevention programmes, in a shift towards predicting disease before it develops. Bupa said it would become the first major private medical insurer to integrate genomics-led prevention into…...
Try GEBRA: A Hands-On Variant Interpretation Experience Try GEBRA: A Hands-On Variant Interpretation Experience
1+ day, 14+ min ago (173+ words) If you have been curious about GEBRA but did not want to start with a demo request, there is now an easier way to explore the platform. The new GEBRA tutorial experience is now live. You can walk through the…...
Mahzi Therapeutics Announces FDA Rare Pediatric Disease Designation for MZ-1866 Investigational Therapy for Pitt Hopkins Syndrome
1+ day, 15+ hour ago (210+ words) Aug 25, 2026, 10:00 ET MZ‑1866 Phase 1/2 UNITE Study is now more than 50% enrolled The MZ-1866 Phase 1/2 study has enrolled 7 of 12 planned participants and enrollment is projected to be completed by the end of this year. MZ-1866 was developed in collaboration with the…...
Bupa genetic risk testing service to go live next week
1+ day, 19+ hour ago (541+ words) Bupa’s DNA-led preventative care services, which aim to help people understand their genetic risk of serious disease, are set to go live next week. The insurer expects 200,000 people will benefit from the overall preventative pathway service by the end of…...
Murdoch Children’s Research Institute gene therapy aims to reverse genetic heart disease | Fox News Video
1+ day, 13+ hour ago (104+ words) Murdoch Children’s Research Institute gene therapy aims to reverse genetic heart disease Fox News Murdoch Children’s Research Institute gene therapy aims to reverse genetic heart disease Murdoch Children’s Research Institute researcher Dr. James McNamara joins ‘Fox & Friends’ to discuss a…...