Evinacumab for treating homozygous familial hypercholesterolemia in children aged 5 to 11 TS ID 11806Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Venglustat for treating gangliosidoses in people 2 years and over ID 6358Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC