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Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study

Dafne D.G. Horovitz, Emília K.E.A. Leão, Erlane M. Ribeiro, Ana M. Martins, Anneliese L. Barth, João I.C.F. Neri, Marcelo Kerstenetzky, Ana C.M. Siqueira, Bethania F.R. Ribeiro, Chong A. Kim, Francisca C. Santos, José F.S. Franco, Leniza C.L. Lichtvan, Liane R. Giuliani, Maria Do C.S. Rodrigues, Renata C.F. Bonatti, Thaís B. Teixeira , Alexandra Gonçalves, Charles M. Lourenço, Ane S.S. Pereira, Angelina X. Acosta

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Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study

Angela Schulz, MD PhD , Nicola Specchio, MD PhD, Emily de los Reyes, MD, Paul Gissen, MD PhD, Miriam Nickel, MD, Marina Trivisano, MD PhD, Shawn C Aylward, MD, Anupam Chakrapani, MD, Christoph Schwering, MD, Eva Wibbeler, MD, Lena Marie Westermann, MD, Douglas J Ballon, PhD, Jonathan P Dyke, PhD DABMP, Anu Cherukuri, PhD, Shailesh Bondade, MD, Peter Slasor, ScD, Jessica Cohen Pfeffer, MD

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Management of early treated adolescents and young adults with phenylketonuria: Development of international consensus recommendations using a modified Delphi approach

Barbara K. Burton, Álvaro Hermida, Amaya Bélanger-Quintana, Heather Bell, Kendra J. Bjoraker, Shawn E. Christ, Mitzie L. Grant, Cary O. Harding, Stephan C.J. Huijbregts, Nicola Longo, Markey C. McNutt II, Mina D. Nguyen-Driver, André L. Santos Pessoa, Júlio César Rocha, Stephanie Sacharow, Amarilis Sanchez-Valle, H. Serap Sivri, Jerry Vockley, Mark Walterfang, Sarah Whittle, Ania C. Muntau