Listar por autor "Wasserstein, Melissa P."
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Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
Jones, Simon A.; McGovern, Margaret M.; Lidove, Olivier; Giugliani, Roberto; Mistry, Pramod; Vici, Carlo Dionisi; Munõz Rojas, Maria Verônica; Nalysnyk, Lubomyra; Schecter, Alison D.; Wasserstein, Melissa P. (2020) [Artículo de periódico]Background: Acid sphingomyelinase deficiency (ASMD) also known as Niemann-Pick disease, is a rare lysosomal storage disorder with a diverse disease spectrum that includes slowly progressive, chronic visceral (type B) and ... -
Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency
McGovern, Margaret M.; Vici, Carlo Dionisi; Giugliani, Roberto; Hwu, Paul; Lidove, Olivier; Lukacs, Zoltan; Mengel, Karl Eugen; Mistry, Pramod; Schuchman, Edward; Wasserstein, Melissa P. (2017) [Artículo de periódico]Background: Acid sphingomyelinase deficiency (ASMD) is a rare, progressive, and often fatal lysosomal storage disease. The underlying metabolic defect is deficiency of the enzyme acid sphingomyelinase that results in ... -
A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults : one-year results
Wasserstein, Melissa P.; Giugliani, Roberto; Kumar, Monica (2022) [Artículo de periódico]Purpose: This trial aimed to assess the efficacy and safety of olipudase alfa enzyme replacement therapy for non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adults. Methods: A phase ...