1. A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy. (30th April 2019) Authors: Lochmüller, Hanns; Behin, Anthony; Caraco, Yoseph; Lau, Heather; Mirabella, Massimiliano; Tournev, Ivailo; Tarnopolsky, Mark; Pogoryelova, Oksana; Woods, Catherine; Lai, Alexander; Shah, Jinay; Koutsoukos, Tony; Skrinar, Alison; Mansbach, Hank; Kakkis, Emil; Mozaffar, Tahseen Journal: Neurology Issue: Volume 92:Number 18(2019) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial. Issue 10189 (15th June 2019) Authors: Imel, Erik A; Glorieux, Francis H; Whyte, Michael P; Munns, Craig F; Ward, Leanne M; Nilsson, Ola; Simmons, Jill H; Padidela, Raja; Namba, Noriyuki; Cheong, Hae Il; Pitukcheewanont, Pisit; Sochett, Etienne; Högler, Wolfgang; Muroya, Koji; Tanaka, Hiroyuki; Gottesman, Gary S; Biggin, Andrew; Perwa... Journal: Lancet Issue: Volume 393:Issue 10189(2019) Page Start: 2416 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Development and preliminary evidence of the psychometric properties of the GNE myopathy functional activity scale. (April 2018) Authors: Mayhew, Jill; Bonner, Nicola; Arbuckle, Rob; Turnbull, Alice; Bowden, Alexandra; Skrinar, Alison Journal: Journal of comparative effectiveness research Issue: Volume 7:Number 4(2018) Page Start: 381 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Efficacy and safety of burosumab in children aged 1–4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial. Issue 3 (March 2019) Authors: Whyte, Michael P; Carpenter, Thomas O; Gottesman, Gary S; Mao, Meng; Skrinar, Alison; San Martin, Javier; Imel, Erik A Journal: Lancet Issue: Volume 7:Issue 3(2019) Page Start: 189 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗