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Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease

Deborah Elstein, Atul Mehta, Derralynn A Hughes, Pilar Giraldo, Joel Charrow, Laurie Smith, Suma P Shankar, Thomas N Hangartner, Yune Kunes, Nan Wang, Eric Crombez, Ari Zimran

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Correction of enzyme levels with allogeneic hematopoeitic progenitor cell transplantation in Niemann‐Pick type B

Jennifer Schneiderman, Kimberly Thormann, Joel Charrow, Morris Kletzel

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Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Ans T van der Ploeg, Richard Barohn, Lisa Carlson, Joel Charrow, Paula R Clemens, Robert J Hopkin, Priya S Kishnani, Pascal Laforêt, Claire Morgan, Sharon Nations, Alan Pestronk, Horacio Plotkin, Barry E Rosenbloom, Katherine B Sims, Elisa Tsao

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Paediatric non-neuronopathic Gaucher disease: recommendations for treatment and monitoring

Antonio Baldellou, Generoso Andria, Pauline E Campbell, Joel Charrow, Ian J Cohen, Gregory A Grabowski, Chris M Harris, Paige Kaplan, Kieran McHugh, Eugen Mengel, Ashok Vellodi

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Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry.

C M Eng, J Fletcher, W R Wilcox, S Waldek, C R Scott, D O Sillence, F Breunig, J Charrow, D P Germain, K Nicholls, M Banikazemi

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Genetics professionals' opinions of whole-genome sequencing in the newborn period.

Elizabeth Ulm, W Gregory Feero, Richard Dineen, Joel Charrow, Catherine Wicklund

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Long-term treatment outcomes in Gaucher disease.

Joel Charrow, C Ronald Scott

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Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase

Ari Zimran, Gregory M Pastores, Anna Tylki-Szymanska, Derralynn A Hughes, Deborah Elstein, Rebecca Mardach, Christine Eng, Laurie Smith, Margaret Heisel-Kurth, Joel Charrow, Paul Harmatz, Paul Fernhoff, William Rhead, Nicola Longo, Pilar Giraldo, Juan A Ruiz, David Zahrieh, Eric Crombez, Gregory A Grabowski

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Uncombable hair (pili trianguli et Canaliculi): Evidence for dominant inheritance with complete penetrance based on scanning electron microscopy

A A Hebert, J Charrow, N B Esterly, D F Fretzin

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Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease

Correction of enzyme levels with allogeneic hematopoeitic progenitor cell transplantation in Niemann‐Pick type B

Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Paediatric non-neuronopathic Gaucher disease: recommendations for treatment and monitoring

Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry.

Genetics professionals' opinions of whole-genome sequencing in the newborn period.

Long-term treatment outcomes in Gaucher disease.

Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase

Uncombable hair (pili trianguli et Canaliculi): Evidence for dominant inheritance with complete penetrance based on scanning electron microscopy

Growth Hormone Excess in Children with Neurofibromatosis Type 1-Associated and Sporadic Optic Pathway Tumors

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease

Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease

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