Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults.

Lachmann, Robin H; Diaz, George A; Wasserstein, Melissa P; Armstrong, Nicole M; Yarramaneni, Abhimanyu; Kim, Yong; Kumar, Monica

Lachmann, Robin H; Diaz, George A; Wasserstein, Melissa P; Armstrong, Nicole M; Yarramaneni, Abhimanyu; Kim, Yong; Kumar, Monica.

Afiliación

Lachmann RH; National Hospital for Neurology, University College London Hospitals, London, UK. r.lachmann@nhs.net.
Diaz GA; Icahn School of Medicine at Mount Sinai, New York, NY, US.
Wasserstein MP; Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, US.
Armstrong NM; Sanofi, Cambridge, MA, USA.
Yarramaneni A; Sanofi, Bridgewater, NJ, USA.
Kim Y; Sanofi, Paris, France.
Kumar M; Sanofi, Bridgewater, NJ, USA.

Orphanet J Rare Dis ; 18(1): 94, 2023 04 25.

Article en En | MEDLINE | ID: mdl-37098529

Asunto(s)

Enfermedad de Niemann-Pick Tipo A; Enfermedades de Niemann-Pick; Adulto; Humanos; Terapia de Reemplazo Enzimático; Lípidos/uso terapéutico; Esfingomielina Fosfodiesterasa/uso terapéutico

Palabras clave

Acid sphingomyelinase deficiency; Niemann-Pick disease type B and type A/B; Olipudase alfa; Recombinant human acid sphingomyelinase

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades de Niemann-Pick / Enfermedad de Niemann-Pick Tipo A Límite: Adult / Humans Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2023 Tipo del documento: Article País de afiliación: Reino Unido

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