TOULOUSE, France – ABIONYX Pharma (FR0012616852 – ABNX – PEA PME eligible), a new generation biotech company dedicated to innovative therapies for patients, today announces positive clinical results from CER-001 in the LCAT (lecithin-cholesterol acyltransferase) deficiency disease published in the Annals of Internal Medicine. The patient with inherited mutations in the lecithin-cholesterol...
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PARIS, FRANCE – Abivax SA (Euronext Paris:ABVX))(NASDAQ:ABVX), a clinical-stage biotechnology company focused on developing therapeutics that harness the body’s natural regulatory mechanisms to stabilize the immune response in patients with chronic inflammatory diseases, today announced that four scientific abstracts on its lead drug candidate, obefazimod for the treatment of moderately to...
LUND, Sweden — Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced a positive outcome of the interim analysis for FALCON, the potentially registrational study evaluating KL1333 in patients with primary mitochondrial disease. The study evaluates fatigue...
Patient registries collect information about the health status of people with a certain disease. They are particularly important for rare diseases such as Lambert-Eaton myasthenic syndrome (LEMS), bringing together a small patient population and gathering information about how the disease affects them. Researchers in academia and pharmaceutical companies can then use this...
SAN DIEGO, Calif. – Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved DAYBUE® STIX (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients two years of age and...
– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030) – Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate...
SAN DIEGO, Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia’s submission. “Rett syndrome is a profoundly debilitating and complex...
SAN DIEGO. Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the journal Developmental Medicine and Child Neurology published interim results from the caregiver-reported observational online, open-label, ongoing LOTUS study evaluating effectiveness and tolerability outcomes in patients with Rett syndrome who are prescribed DAYBUE® (trofinetide) under routine clinical care in the U.S. Findings included reported...
According to the results of a phase 1/2 study, the Bruton tyrosine kinase (BTK) inhibitor acalabrutinib was safe and efficacious, with durable remissions, in patients with previously untreated chronic lymphocytic leukemia (CLL). The findings were reported in Blood. The single-arm study, ACE-CL-001 (ClinicalTrials.gov Identifier: NCT02029443), evaluated efficacy and safety of acalabrutinib monotherapy...
Acasti Pharma Inc., a late-stage, specialty pharma company advancing three clinical stage drug candidates addressing rare and orphan diseases, recognizes and celebrates Rare Disease Day (February 28), as established by the European Organization for Rare Diseases.