International Niemann–Pick Disease Alliance

Sanofi Genzyme: ASCEND and ASCEND-Peds Studies Complete Enrollment

Sanofi Genzyme’s Global Project Team would like to announce that our pivotal registration study (ASCEND Study) for adult patients with chronic, systemic acid sphingomyelinase deficiency (ASMD) Types B and A/B (or intermediate type) and our pediatric study (ASCEND-Peds) also treating Type B and A/B ASMD pediatric patients have successfully completed enrollment for the targeted number of patients for each of these trials. These clinical trials were initiated by Sanofi Genzyme for adults and pediatric ASMD patients without the severe neurologic phenotype in 2015 and in 2016, respectively. Patients in these trials will be assessed one year after enrollment.

Sanofi Genzyme would like to express its profound gratitude to the patients and their families who participated in these trials to enable further development of an investigational therapy for this devastating ultra-rare disease.  Sanofi Genzyme could not have achieved this important milestone without your selfless participation and support.



Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

Think Again. Think NP-C

Join our international campaign to improve diagnosis of NP-C!