Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance.
Mehmet Umut AkyolTord D AldenHernan AmartinoJane AshworthKumar BelaniKenneth I BergerAndrea BorgoElizabeth BraunlinYoshikatsu EtoJeffrey I GoldAndrea JesterSimon A JonesCengiz KarsliWilliam MackenzieDiane Ruschel MarinhoAndrew McFadyenJim McGillJohn J MitchellJoseph MuenzerTorayuki OkuyamaPaul J OrchardBob StevensSophie ThomasRobert WalkerRobert WynnRoberto GiuglianiPaul HarmatzChristian J HendrikszMaurizio Scarpanull nullnull nullPublished in: Orphanet journal of rare diseases (2019)
This manuscript provides evidence- and consensus-based recommendations for the management of patients with MPS IVA and is for use by healthcare professionals that manage the holistic care of patients with the intention to improve clinical- and patient-reported outcomes and enhance patient quality of life. It is recognised that the guidance provided represents a point in time and further research is required to address current knowledge and evidence gaps.