Nitisinone for treating alkaptonuria [ID2691]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease in people 12 to 17 years TS ID 11887Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
Prolonged-release diazoxide choline for treating Prader-Willi syndrome [ID1389]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC