Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Carla Hollak"'
Autor:
Melissa P. Wasserstein, Robin Lachmann, Carla Hollak, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto Bernardo Guelbert, Julia B. Hennermann, Takayuki Ikezoe, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Abhimanyu Yarramaneni, Nicole M. Armstrong, Yong Kim, Monica Kumar
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-12 (2023)
Abstract Background Olipudase alfa is a recombinant human acid sphingomyelinase enzyme replacement therapy for non-central-nervous-system manifestations of acid sphingomyelinase deficiency (ASMD). The ASCEND randomized placebo-controlled trial in adu
Externí odkaz:
https://doaj.org/article/7737861ecbf54c38965ac8ebac64bc3c
Autor:
Tarekegn Geberhiwot, Melissa Wasserstein, Subadra Wanninayake, Shaun Christopher Bolton, Andrea Dardis, Anna Lehman, Olivier Lidove, Charlotte Dawson, Roberto Giugliani, Jackie Imrie, Justin Hopkin, James Green, Daniel de Vicente Corbeira, Shyam Madathil, Eugen Mengel, Fatih Ezgü, Magali Pettazzoni, Barbara Sjouke, Carla Hollak, Marie T. Vanier, Margaret McGovern, Edward Schuchman
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-28 (2023)
Abstract Background Acid Sphingomyelinase Deficiency (ASMD) is a rare autosomal recessive disorder caused by mutations in the SMPD1 gene. This rarity contributes to misdiagnosis, delayed diagnosis and barriers to good care. There are no published nat
Externí odkaz:
https://doaj.org/article/3b60746a318b45afbe6b8545bdc098d5
Autor:
Melissa Wasserstein, Robin Lachmann, Carla Hollak, Laila Arash-Kaps, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto Bernardo Guelbert, Takayuki Ikezoe, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Eubekir Senates, Michel Tchan, Jesus Villarrubia, Yixin Chen, Sandy Furey, Beth L. Thurberg, Atef Zaher, Monica Kumar
Publikováno v:
Genetics in medicine, 24(7), 1425-1436. Lippincott Williams and Wilkins
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 2/3, 52 week, internationa
Publikováno v:
International Journal of Technology Assessment in Health Care. 38:S89-S90
IntroductionBefore admission to the insured package, the price of a medicine is usually assessed on the basis of the value of the medicine for the patient: the effect size on health and survival must be in line with the costs. That seems like a fair
Autor:
Carla Hollak
Publikováno v:
Physician's Guide to the Diagnosis, Treatment, and Follow-Up of Inherited Metabolic Diseases ISBN: 9783030677268
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::1c3303bc675e61a867f30f43f9e9fa68
https://doi.org/10.1007/978-3-030-67727-5_60
https://doi.org/10.1007/978-3-030-67727-5_60
Autor:
Melissa Wasserstein, Antonio Barbato, Renata Gallagher, Roberto Giugliani, Norberto Guelbert, Julia Hennermann, Carla Hollak, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth Thurberg, Abhimanyu Yarramaneni, Andreea Rawlings, Yong Kim, Monica Kumar
Publikováno v:
Genetics in Medicine. 24:S176-S177
Publikováno v:
Journal of Clinical Oncology. 40:1586-1586
1586 Background: After European Medicines Agency Marketing Authorization (EMA-MA) different national reimbursement processes may contribute to unequal access throughout the EU. The aim of this study is to investigate the access time to new anticancer
Autor:
Melissa P. Wasserstein, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto B. Guelbert, Julia B. Hennermann, Carla Hollak, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Abhimanyu Yarramaneni, Andreea M. Rawlings, Yong Kim, Monica Kumar
Publikováno v:
Molecular Genetics and Metabolism. 135:S126-S127
Autor:
Robin Lachmann, Carla Hollak
Publikováno v:
Journal of inherited metabolic disease, 41(5), 899-900. Springer Netherlands
Autor:
Carla Hollak
Publikováno v:
Dr. Pangloss en de geneeskunde
Deze wereld is de beste van alle mogelijke werelden. Dit door dr. Pangloss uitgedragen onwrikbare geloof en vertrouwen in een positieve uitkomst is onmiskenbaar doorgedrongen tot de geneeskunde. De ontwikkelingen in de moleculaire biologie, biochemie
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::c573c514d9b81360e7228015990bba33
https://doi.org/10.5117/9789056296230
https://doi.org/10.5117/9789056296230