Hinxton, England – For the first time, scientists have systematically analysed somatic mutations in stomach lining tissue to unpick mutational processes, some of which can lead to cancer. The team also uncovered hints of a potential new cause of stomach cancer that needs further research. Researchers at the Wellcome Sanger...
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Nashville, Tennessee – Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD). The therapy, nonmyeloablative haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy), is proving to have equivalent efficacy...
Amanda Hamacher was in grade school when she began to lose her sight and hearing as a result of a rare genetic disorder called Alstrom syndrome. Now, the 21-year-old cannot see and has severe hearing loss. Yet, she enjoys a newfound sense of freedom thanks to a program sponsored by...
Intestinal permeability and an overgrowth of bacteria in the small intestine are both associated with nonalcoholic fatty liver disease (NAFLD). These findings are revealed in a new study in the June issue of Hepatology, a journal published by John Wiley & Sons on behalf of the American Association for the...
AUSTIN, Texas — IntraBio Inc. today announced positive results from the full data set for the pivotal Phase III IB1001-303 clinical trial, “Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, Placebo-Controlled, Double-Blind, Crossover Study” (NCT06673056) evaluating N-acetyl-L-leucine (levacetylleucine) in pediatric and adult patients with Ataxia-Telangiectasia (A-T). Key Findings Efficacy...
AUSTIN, TX – IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients...
AUSTIN, Texas — IntraBio Inc. today announced positive topline results from its pivotal Phase III IB1001-303 clinical trial, “Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, Placebo-Controlled, Double-Blind, Crossover Study” (NCT06673056) evaluating N-acetyl-L-leucine (levacetylleucine) in pediatric and adult patients with Ataxia-Telangiectasia (A-T). The primary endpoint of the trial evaluated the...
AUSTIN, Texas — IntraBio Inc. today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T), a rare, progressive, inherited neurodegenerative disorder. This submission represents the first regulatory application submitted to the U.S. Food...
AUSTIN, Texas — IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA® (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) disease, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). AQNEURSA® is approved...
AUSTIN, Texas — IntraBio Inc., a global biopharmaceutical company developing therapies for neurological diseases, today announced that its pivotal Phase III trial of levacetylleucine in patients with CACNA1A-related disorders has been authorized to proceed across all participating regions, including the U.S., the U.K., the European Union, and Switzerland. Twelve multinational...