Open-Label Extension Study Testing Arimoclomol in Patients With NPC +++ +++ +

CLINICAL PERSPECTIVES Open-Label Extension Study Testing Arimoclomol in Patients With NPC https://checkrare.com/open-label-extension-study-testing-arimoclomol-in-patients-with-npc/ A recent paper published in Molecular Genetics and Metabolism presented safety and efficacy outcomes from the 48-month open-label extension of the NPC-002 study of arimoclomol in patients with Niemann-Pick type C (NPC). Post Hoc Analysis of Long-Term Efficacy of Migalastat in Females With Fabry Disease https://checkrare.com/post-hoc-analysis-of-long-term-efficacy-of-migalastat-in-females-with-fabry-disease/ A recent post hoc analysis of long-term efficacy of migalastat in female patients with Fabry disease was published in the Journal of Medical Genetics. Current Trends in the Healthcare Job Market https://checkrare.com/current-trends-in-the-healthcare-job-market/ Vicki Salemi, Career Expert, discusses current trends in the healthcare job market and expectations for 2026. Efficacy of Pozelimab + Cemdisiran To Treat Paroxysmal Nocturnal Hemoglobinuria https://checkrare.com/efficacy-of-pozelimab-cemdisiran-to-treat-paroxysmal-nocturnal-hemoglobinuria/ Jun Ho Jang, MD, PhD, Professor, Division of Hematology-Oncology at Samsung Medical Center, discusses results from an open-label extension study testing pozelimab plus cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH), who previously received the combination therapy or received ravulizumab. Results from the ElevAATe Clinical Trial of Efdoralprin Alfa for Patients With AAT Deficiency https://checkrare.com/results-from-the-elevaate-clinical-trial-of-efdoralprin-alfa-for-patients-with-aat-deficiency/ Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the ElevAATe clinical trial of efdoralprin alfa for the treatment of patients with alpha-1 antitrypsin deficiency (AATD). Ipsen’s Current Rare Disease Therapies: Approved and In Development https://checkrare.com/ipsens-current-rare-disease-therapies-approved-and-in-development/ Christelle Huguet, PhD, Head of Research and Development at Ipsen, discusses the company’s current approved orphan drugs and those in development for rare diseases. Results From the PEGASUS Clinical Trial of Pegvaliase in Patients With PKU https://checkrare.com/results-from-the-pegasus-clinical-trial-of-pegvaliase-in-patients-with-pku/ Kevin Eggan, PhD, Chief Scientific Officer at BioMarin, discusses new results from the PEGASUS clinical trial of Palynziq (pegvaliase) for treating patients with phenylketonuria (PKU).