Florian Eichler News Today

- Following treatment, the N-acetylaspartate levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s... | May 22, 2023

The groundbreaking treatment is one of six gene therapies cleared since 2017 that have transformed the outlook for disorders once considered hopeless — and also renewed questions about how the health care system can afford such breathtakingly expensive medicines.

Guangping Gao, PhD, will be honored by the National Tay-Sachs & Allied Diseases Association for his work in identifying the Canavan gene, revitalizing gene therapy, and his many accomplishments leading to potential treatments for Canavan disease.

POXEL SA (Euronext POXEL FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis

/PRNewswire/ -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced...

BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Canavan Disease - read this article along with other careers information, tips and advice on BioSpace

An experimental gene therapy was little Alissas 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 minu...

Message : Required fields 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...

Share: First Data Presented Showcasing Autobahns 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 a...

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, t...