Sanofi Genzyme and PerkinElmer Genomics have launched the Lantern Project, a comprehensive genetic testing program that aims to overcome the barriers to diagnosing rare diseases, such as Pompe disease. Rare, inherited disorders can be quite challenging to diagnose because they often are mistaken for more common diseases,…
Sanofi Genzyme
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
Results of a Phase 4 clinical trial support the effectiveness of Lumizyme (4,000 liters recombinant human GAA) for 52 weeks in maintaining clinical stability in Pompe disease patients previously treated with the therapy’s 160-liter formulation, called Myozyme. The study, “Efficacy, safety profile, and immunogenicity of alglucosidase alfa…