Genzyme Clinical Trial Update ~ Safety and Tolerability of Within-Patient Dose Escalation
Dear NNPDF Families and Friends,
During the 2014 Annual Meeting of the American Society of Human Genetics (ASHG), held from October 18th-22nd in San Diego, California, Dr. Melissa Wasserstein M.D. was honored to give a presentation on the results of the safety and tolerability of the enzyme replacement therapy (ERT) 1b clinical trial being funded by Genzyme, a Sanofi Company.
The key information noted within the abstract was that: “The dose escalation regimen was well tolerated, with all patients reaching the maximum dose of 3.0 mg/kg. No serious or severe adverse events or deaths were reported. Related AEs consisted predominantly of infusion-associated reactions, the majority of which were mild and resolved without sequalae. A positive response to treatment with rhASM was observed in liver sphingomyelin content and several exploratory efficacy parameters, including spleen and liver volumes, pulmonary function testing, lung imaging, lipid profile, and quality of life assessments.”