A foreign drugmaker has thrown a gauntlet to the Korean rare disease treatment market. Italy-based Recordati is the company. On Rare Disease Day Feb. 28, the company launched Recordati Korea, its Korean offshoot and Asia-Pacific headquarters, declaring its official advance to the Korean and regional market. Unlike most other pharma...
treatment News
Toruń, Poland – Boys suffering from Barth syndrome, a genetic determined disease with a very high mortality rate, finally have hope for a cure. What has contributed to this finding is research of Dr. habil Karolina Mikulska-Ruminska, Nicolaus Copernicus University (Torun, Poland) CU Professor from the Institute of Physics. The...
Two biotechs are shoring up their pipelines and commercial aspirations, tacking on a clinical-stage rare disease drug and a preclinical cancer med, respectively. Fortress Biotech-founded Avenue Therapeutics is adding on a phase 1/2-stage asset from AnnJi Pharmaceutical for the treatment of spinal and bulbar muscular atrophy (SBMA). Financial details are...
Edmonton, Canada – A University of Alberta team has developed a new step to improve the process for creating insulin-producing pancreatic cells from a patient’s own stem cells, bringing the prospect of injection-free treatment closer for people with diabetes. The researchers take stem cells from a single patient’s blood and...
SHANGHAI — Abbisko Therapeutics Co., Ltd. (“Abbisko Therapeutics” hereafter) announced that two major clinical updates of its CSF-1R inhibitor pimicotinib（ABSK021）were presented at the 2023 Connective Tissue Oncology Society Annual Meeting, which is held in Ireland from November 1 to 4, 2023. The two clinical updates include reporting the design of...
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...
– 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...
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.
CDER’s Accelerating Rare disease Cures (ARC) Program harnesses CDER’s collective expertise and activities to provide strategic overview and coordination of CDER’s rare disease activities. CDER’s ARC Program is governed by leadership from across CDER’s Office of the Center Director, Office of New Drugs, and the Office of Translational Sciences. The program...