Boston, MA – A new international consensus report on precision medicine in diabetes prevention and care highlights the significant advancements in precision medicine in diabetes prevention, diagnosis, treatment, and prognosis while also shedding light on numerous knowledge gaps. The report, Second international consensus report on gaps and opportunities for the...
treatment News
FRAMINGHAM, Mass. & SALISBURY, England — KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the newly published International Guideline on the Diagnosis and Management of Pediatric Patients With Hereditary Angioedema recommends EKTERLY® (sebetralstat) as a first-line therapy for the acute treatment of hereditary angioedema (HAE) attacks in adolescents aged 12...
Barcelona, Spain – An international team of scientists led by ICREA researcher and Director of the Life Sciences Department at the Barcelona Supercomputing Centre – Centro Nacional de Supercomputación (BSC-CNS), Alfonso Valencia, has developed a technology based on artificial intelligence (AI) for the study of orphan diseases and has successfully...
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...
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...