CAMBRIDGE, Mass. — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that longer-term data from the ongoing Phase 1 trial of investigational nex-z for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) will be presented at the 5th International...
treatment News
MORRISTOWN, N.J. — Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA)...
Sendai, Japan – An interim analysis of an ongoing Japanese study published in Modern Rheumatology demonstrates that mepolizumab is safe and effective in improving disease control among patients with eosinophilic granulomatosis with polyangiitis (EGPA), a form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). Remission is typically achieved and maintained in...
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...
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...
Trento, Italy – Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, is one of the most devastating neurodegenerative disorders. By progressively destroying the motor neurons that control voluntary muscle movement, ALS gradually robs patients of their ability to walk, speak, swallow, and eventually breathe. While multidisciplinary care has...
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...