• Leadership Profile

Brinda Balakrishnan

• Publication

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Christian J. Hendriksz, Barbara Burton, Thomas R. Fleming, Paul Harmatz, Derralynn Hughes, Simon A. Jones, Shuan-Pei Lin, Eugen Mengel, Maurizio Scarpa, Vassili Valayannopoulos, Roberto Giugliani, STRIVE Investigators, Peter Slasor, Debra Lounsbury, Wolfgang Dummer

• Publication

Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance

Mehmet Umut Akyol1, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz , Maurizio Scarpa, MPS Consensus Programme Steering Committee and MPS Consensus Programme Co-Chairs

• Publication

Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance

Mehmet Umut Akyol, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz, Maurizio Scarpa, MPS Consensus Programme Steering Committee and MPS Consensus Programme Co-Chairs

• Publication

Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients

Sara E. Mole, Angela Schulz, Eben Badoe, Samuel F. Berkovic, Emily C. de Los Reyes, Simon Dulz, Paul Gissen, Norberto Guelbert, Charles M. Lourenco, Heather L. Mason, Jonathan W. Mink, Noreen Murphy, Miriam Nickel, Jofre E. Olaya, Maurizio Scarpa, Ingrid E. Schefer, Alessandro Simonati, Nicola Specchio, Ina Von Löbbecke, Raymond Y. Wang and Ruth E. Williams

• Publication

Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria

Nicola Longo, MD, PhD, David Dimmock, MD, Harvey Levy, MD, Krista Viau, PhD, RD, Heather Bausell, RD, LDN, Deborah A. Bilder, MD, Barbara Burton, MD, Christel Gross, RN, Hope Northrup, MD, Fran Rohr, MS, RD, Stephanie Sacharow, MD, Amarilis Sanchez-Valle, MD, Mary Stuy, MD, Janet Thomas, MD, Jerry Vockley, MD, PhD, Roberto Zori, MD and Cary O. Harding, MD

• Publication

Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics

Darius Adams, Hans C. Andersson, Heather Bausell, Kea Crivelly, Caroline Eggerding, Melissa Lah, Joshua Lilienstein, Kristin Lindstrom, Markey McNutt, Joseph W. Ray, Heather Saavedra, Stephanie Sacharow, Danielle Starin, Jennifer Tiffany-Amaro, Janet Thomas, Erika Vucko, Leah B. Wessenberg, Kaleigh Whitehall

• Page

Home

• Page

Research & Early Development Postdoctoral Fellowship Program

• Page

Web Request Form

• Page

Web Request Form – IT

• Page

Web Request Form – DE

• Page

Web Request Form – FR

• Page

Web Request Form – RU

• Page

Web Request Form – TU

• Page

Web Request Form – JP