An adult with acid sphingomyelinase deficiency type B has hepatosplenomegaly and progressive restrictive lung disease but no central nervous system in...

Question

An adult with acid sphingomyelinase deficiency type B has hepatosplenomegaly and progressive restrictive lung disease but no central nervous system involvement. They have never received olipudase alfa. What is the current NICE commissioning position?

Accepted Answer

Do not start olipudase alfa through routine NHS commissioning

Answer

Start olipudase alfa routinely because type B is within its licence

Answer

Start only after neurological involvement develops

Answer

Start through a mandatory managed-access agreement for every adult

Answer

Start only after failure of miglustat and eliglustat

Perioperative Anticoagulation — MRCP Part 1 MCQ

An adult with acid sphingomyelinase deficiency type B has hepatosplenomegaly and progressive restrictive lung disease but no central nervous system involvement. They have never received olipudase alfa. What is the current NICE commissioning position?