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Treatabolome project designed to shorten diagnosis-to-treatment time for patients with rare diseases

The Treatabolome project is a research initiative to develop a freely available, interoperable online platform to disseminate rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise to reduce treatment delays. This initiative is highly relevant to neuromuscular disorders as they are rare diseases by definition. In this special issue of the Journal of Neuromuscular Diseases, experts contribute Treatabolome-feeding systematic literature reviews on rare neurological and neuromuscular disorders. ....

Noord Holland , France General , European Research , European Reference Networks , Centre De Recherche En Myologie , Research Project , Neuromuscular Diseases , Guest Editor Gis , Sorbonne Universit , Institut De Myologie , Centre De Recherche , Rare Neurological Diseases , Rare Neuromuscular Diseases , Genome Phenome Analysis Platform , Reference Networks , Medicine Health , நூற்த் ஹாலண்ட் , பிரான்ஸ் ஜநரல் , ஐரோப்பிய ஆராய்ச்சி , ஐரோப்பிய குறிப்பு நெட்வொர்க்குகள் , ஆராய்ச்சி ப்ராஜெக்ட் , நரம்புத்தசை நோய்கள் , மையம் டி ரெக்ஃப்‌ச் , ரேர் நரம்பியல் நோய்கள் , குறிப்பு நெட்வொர்க்குகள் , மருந்து ஆரோக்கியம் ,

New initiative to reduce treatment delays for patients with rare diseases

The Treatabolome project is a research initiative to develop a freely available, interoperable online platform dedicated to disseminating rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise. ....

France General , Emily Henderson , European Research , European Reference Networks , Centre De Recherche En Myologie , Research Project , Neuromuscular Diseases , Guest Editor , Sorbonne Universit , Institut De Myologie , Centre De Recherche , Rare Neurological Diseases , Rare Neuromuscular Diseases , Genome Phenome Analysis Platform , Reference Networks , Rare Disease , Research Project , பிரான்ஸ் ஜநரல் , எமிலி ஹென்டர்சன் , ஐரோப்பிய ஆராய்ச்சி , ஐரோப்பிய குறிப்பு நெட்வொர்க்குகள் , ஆராய்ச்சி ப்ராஜெக்ட் , நரம்புத்தசை நோய்கள் , விருந்தினர் ஆசிரியர் , மையம் டி ரெக்ஃப்‌ச் , ரேர் நரம்பியல் நோய்கள் ,

Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)

Published: Feb 03, 2021
- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies
- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021
NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease. ....

University Of Massachusetts Medical School , United States , Paragv Meswani , Florian Eichler , Terencer Flotte , Tay Sachs Sandhoff , Josephine Belluardo , Tay Sachs , Gavin Corcoran , Leukodystrophy Service Of The Center , Exchange Commission On , Sio Gene Therapies Inc , National Tay Sachs Allied Diseases Association , Exchange Commission , Lifesci Communications , Gene Therapies , Chief Rd Officer , Leukodystrophy Service , Rare Neurological Diseases , Massachusetts General Hospital , Orphan Drug , Rare Pediatric Disease Designation , Massachusetts Medical School , Executive Director , National Tay Sachs , Allied Diseases Association ,