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An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future.

An experimental gene therapy was little Alissa s only hope. Now, instead of certain death, she faces an uncertain future.
Karen Weintraub, USA TODAY
© Feldborg family photo
Alissa Feldborg
BOSTON Thomas Feldborg and Daria Rokina set off nearly every afternoon to explore this city. They leave from their hotel in the Charlestown neighborhood, pushing their baby carriage, some days heading deep into downtown, others choosing a path along the Charles River.
Every few minutes, Rokina stops to peek inside and check on 16-month-old Alissa. She gently rubs Alissa s cheek and coos a few soothing words, making sure the little girl is warm enough in her yellow snowsuit and adjusting her sparkly unicorn earmuffs. ....

United States , University Of Massachusetts , Auburn University , Cummings School , Charles River , Thomas Feldborg , Feldenkrais Method , Pavan Cheruvu , Daria Rokina , Miguel Sena Esteves , Cynthia Tifft , Florian Eichler , Isaac Kohane , Douglas Martin , Matty Wilkinson , Joan Horak , Alissa Feldborg , Terence Flotte , Department Of Biomedical Informatics , Langone Health , National Institutes Of Health , Drug Administration , Harvard Medical School , Auburn University College Of Veterinary Medicine , York University Medical Center , Umass Memorial Medical Center ,

Autobahn Therapeutics : Presents Preclinical, Proof of Concept Data for ABX-002 for the Treatment of Adrenomyeloneuropathy

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First Data Presented Showcasing Autobahn’s Novel FAAH-Mediated Prodrug Approach for Selective Delivery of Thyromimetics to the Brain
Treatment with ABX-002 Leads to Reductions in VLCFA in Brain Tissue and Plasma in AMN Mouse Model
Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced the presentation of preclinical data of ABX-002, a CNS-penetrating prodrug that provides a balanced delivery of LL-340001, a potent and selective thyroid hormone receptor beta (TRβ) agonist, for the potential treatment of adrenomyeloneuropathy (AMN), a progressive and debilitating rare disease. The data will be highlighted in a poster presentation at the American Academy of Neurology Annual Meeting, which is being held virtually from April 17-22, 2021. Posters will be made available at the start of the conference and remain viewable online through the virtual forum for 30 days after ....

United States , Florian Eichler , Brian Stearns , American Academy Of Neurology Annual Meeting , Leukodystrophy Service , Autobahn Therapeutics , American Academy , Neurology Annual Meeting , Massachusetts General Hospital , ஒன்றுபட்டது மாநிலங்களில் , புளோரியன் எய்ச்சிலேர் , பிரையன் ஸ்டேர்ன்ஸ் , அமெரிக்கன் கலைக்கழகம் ஆஃப் நரம்பியல் ஆண்டு சந்தித்தல் , ஆட்டோபான் சிகிச்சை , அமெரிக்கன் கலைக்கழகம் , நரம்பியல் ஆண்டு சந்தித்தல் , மாசசூசெட்ஸ் ஜநரல் மருத்துவமனை ,

Autobahn Therapeutics Presents Preclinical, Proof of Concept Data for ABX-002 for the Treatment of Adrenomyeloneuropathy

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First Data Presented Showcasing Autobahn s Novel FAAH-Mediated Prodrug Approach for Selective Delivery of Thyromimetics to the Brain
Treatment with ABX-002 Leads to Reductions in VLCFA in Brain Tissue and Plasma in AMN Mouse Model
Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced the presentation of preclinical data of ABX-002, a CNS-penetrating prodrug that provides a balanced delivery of LL-340001, a potent and selective thyroid hormone receptor beta (TRβ) agonist, for the potential treatment of adrenomyeloneuropathy (AMN), a progressive and debilitating rare disease. The data will be highlighted in a poster presentation at the American Academy of Neurology Annual Meeting, which is being held virtually from April 17-22, 2021. Posters will be made available at the start of the conference and remain viewable online through the virtual forum for 30 days after the meeting. ....

San Diego , United States , Florian Eichler , Brian Stearns , American Academy Of Neurology Annual Meeting , Leukodystrophy Service , First Data Presented Showcasing Autobahn , Mediated Prodrug Approach , Selective Delivery , Brain Tissue , American Academy , Neurology Annual Meeting , Massachusetts General Hospital , சான் டியாகோ , ஒன்றுபட்டது மாநிலங்களில் , புளோரியன் எய்ச்சிலேர் , பிரையன் ஸ்டேர்ன்ஸ் , அமெரிக்கன் கலைக்கழகம் ஆஃப் நரம்பியல் ஆண்டு சந்தித்தல் , மூளை திசு , அமெரிக்கன் கலைக்கழகம் , நரம்பியல் ஆண்டு சந்தித்தல் , மாசசூசெட்ஸ் ஜநரல் மருத்துவமனை ,

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 ,