Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
Latest Information Update: 17 Sep 2021
At a glance
- Drugs Olipudase alfa (Primary)
- Indications Niemann-Pick disease type A; Niemann-Pick disease type B
- Focus Expanded access; Therapeutic Use
- Sponsors Sanofi
- 23 Mar 2021 New trial record