Morquio Syndrome Drugs

Arms Length Body Publication (Published)
NICE

Dec. 13 2023

Source Page: Velmanase alfa for treating alpha-mannosidosis
Publication Type: Supporting evidence
Document: Appraisal consultation committee papers 1 (PDF 11.2 MB) (webpage)

Found: Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome

Departmental Publication (Transparency)
Home Office

Apr. 24 2023

Found: Usher (deaf - blindness) syndrome, and many metabolic and neurological disorders.

Arms Length Body Publication (Published)
NICE

Jul. 15 2022

Source Page: Velmanase alfa for treating alpha-mannosidosis
Publication Type: Evaluation consultation: 2
Document: Committee papers (PDF 1.52 MB) (webpage)

Found: Burden of disease i n patients with Mor quio A syndrome: results from an international patient-reported

Arms Length Body Publication (Published)
NICE

Apr. 20 2022

Source Page: Elosulfase alfa for treating mucopolysaccharidosis type 4A
Publication Type: Supporting evidence
Document: Evaluation consultation committee papers (PDF 8.66 MB) (webpage)

Found: Query Results #1 'morquio syndrome'/exp OR morquio*:de,ab,ti OR morqio*:de,ab,ti OR morkio*:de,ab

Arms Length Body Publication (Published)
NICE

Nov. 12 2021

Source Page: Elosulfase alfa for treating mucopolysaccharidosis type 4A
Publication Type: Evaluation consultation
Document: Committee papers (PDF 8.66 MB) (webpage)

Found: Query Results #1 'morquio syndrome'/exp OR morquio*:de,ab,ti OR morqio*:de,ab,ti OR morkio*:de,ab

Arms Length Body Publication (Published)
NICE

Nov. 12 2021

Source Page: Elosulfase alfa for treating mucopolysaccharidosis type 4A
Publication Type: Evaluation consultation
Document: Public committee slides (PDF 1.47 MB) (webpage)

Found: sulfatase (GALNS) deficiency Marketing authorisationThe treatment of mucopolysaccharidosis, type IVA ( Morquio

Select Committee

Formal Minutes Apr. 01 2021

Committee: Petitions Committee

Found: petition.parliament.uk/petitions/104559 We need Jeremy Hunt MP to get NHS England to fund Vimizin for Morquio

Arms Length Body Publication (Report)
NHS England

Mar. 11 2021

Source Page: Highly Specialised Services 2019
Document: Highly Specialised Services 2019 (PDF)

Found: It prov ides one -stop care to: assess and detect disease complications; prescribe and monitor drugs

Arms Length Body Publication (Published)
NICE

Dec. 13 2019

Source Page: Elosulfase alfa for treating mucopolysaccharidosis type 4A
Publication Type: Invitation to participate
Document: Final scope (PDF 167 KB) (webpage)

Found: Background Mucopolysaccharidosis type IVA (also known as MPS IVA a nd Morquio A syndrome) is an

Select Committee

ORK0333 - Availability of Orkambi on the NHS inquiry

Written Evidence Mar. 04 2019

Inquiry: Availability of Orkambi on the NHS inquiry
Inquiry Status: Closed
Committee: Health and Social Care Committee (Department: Department of Health and Social Care)

Found: “Cystic Fibrosis: Breakthrough Drugs at Break-the-Bank Prices.”