Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports

por Amato, Dominick, Patterson, Mary Anne
Publicado 2018

Three cases of multi-generational Gaucher disease and colon cancer from an Ashkenazi Jewish family: A lesson for cascade screening

por Hannah-Shmouni, Fady, Amato, Dominick
Publicado 2019

Homozygous N396T mutation in Gaucher disease: Portuguese sisters with markedly different phenotypes

por Kimball, Samantha, Choy, Francis, Zay, Agnes, Amato, Dominick
Publicado 2011

Identification of novel splice site mutation IVS9 + 1(G > A) and novel complex allele G355R/R359X in Type 1 Gaucher patients heterozygous for mutation N370S()

por Hoitsema, Kourtnee, Amato, Dominick, Khan, Aneal, Sirrs, Sandra, Choy, Francis Y.M.
Publicado 2016

A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat

por Remenova, Tatiana, Morand, Olivier, Amato, Dominick, Chadha-Boreham, Harbajan, Tsurutani, Scott, Marquardt, Thorsten
Publicado 2015

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study

por Cox, Timothy M, Amato, Dominick, Hollak, Carla EM, Luzy, Cecile, Silkey, Mariabeth, Giorgino, Ruben, Steiner, Robert D
Publicado 2012

Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

por Weinreb, Neal J., Finegold, David N., Feingold, Eleanor, Zeng, Zhen, Rosenbloom, Barry E., Shankar, Suma P., Amato, Dominick
Publicado 2015

Enzyme replacement therapy with taliglucerase alfa: 36‐month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase

por Pastores, Gregory M., Shankar, Suma P., Petakov, Milan, Giraldo, Pilar, Rosenbaum, Hanna, Amato, Dominick J., Szer, Jeffrey, Chertkoff, Raul, Brill‐Almon, Einat, Zimran, Ari
Publicado 2016

Long‐term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment‐naïve patients with Gaucher disease

por Zimran, Ari, Durán, Gloria, Mehta, Atul, Giraldo, Pilar, Rosenbaum, Hanna, Giona, Fiorina, Amato, Dominick J., Petakov, Milan, Muñoz, Eduardo Terreros, Solorio‐Meza, Sergio Eduardo, Cooper, Peter A., Varughese, Sheeba, Chertkoff, Raul, Brill‐Almon, Einat
Publicado 2016

Occurrence of Amyotrophic Lateral Sclerosis in Type 1 Gaucher Disease

por Oliveira, Lais M., Rastin, Tara, Nimmo, Graeme A.M., Ross, Jay P., Dion, Patrick A., Zhang, Ming, Nevay, Dayna-Lynn, Arkadir, David, Gotkine, Marc, Barnett, Carolina, Shoesmith, Christen L., Zimran, Ari, Rogaeva, Ekaterina A., Zinman, Lorne, Rouleau, Guy A., Gan-Or, Ziv, Amato, Dominick, Kalia, Lorraine V.
Publicado 2021