نتائج البحث - "Carla Hollak"

1

دورية أكاديمية

Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial

المصدر: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-12 (2023)

مصطلحات موضوعية: Recombinant human acid sphingomyelinase, Dose escalation, Organomegaly, Lung diffusing capacity, Acid sphingomyelinase deficiency, Niemann–Pick type B, Medicine

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/1750-1172Test

الوصول الحر: https://doaj.org/article/7737861ecbf54c38965ac8ebac64bc3cTest

View record in DOAJ

النص الكامل

2

دورية أكاديمية

Consensus clinical management guidelines for acid sphingomyelinase deficiency (Niemann–Pick disease types A, B and A/B)

المصدر: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-28 (2023)

مصطلحات موضوعية: Acid sphingomyelinase deficiency, ASMD, Niemann–Pick disease, Niemann–Pick disease-a,b,a/b, Guidelines, Diagnosis, Medicine

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/1750-1172Test

الوصول الحر: https://doaj.org/article/3b60746a318b45afbe6b8545bdc098d5Test

View record in DOAJ

النص الكامل

3

دورية أكاديمية

Additional file 1 of Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial

مصطلحات موضوعية: Biochemistry, Medicine, Physiology, Pharmacology, Immunology, Cancer, Recombinant human acid sphingomyelinase, Dose escalation, Organomegaly, Lung diffusing capacity, Acid sphingomyelinase deficiency, Niemann–Pick type B, Niemann–Pick type A/B

العلاقة: https://figshare.com/articles/journal_contribution/Additional_file_1_of_Continued_improvement_in_disease_manifestations_of_acid_sphingomyelinase_deficiency_for_adults_with_up_to_2_years_of_olipudase_alfa_treatment_open-label_extension_of_the_ASCEND_trial/24716926Test

الإتاحة: https://doi.org/10.6084/m9.figshare.24716926.v1Test
https://figshare.com/articles/journal_contribution/Additional_file_1_of_Continued_improvement_in_disease_manifestations_of_acid_sphingomyelinase_deficiency_for_adults_with_up_to_2_years_of_olipudase_alfa_treatment_open-label_extension_of_the_ASCEND_trial/24716926Test

View record in BASE

4

A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results

المساهمون: Endocrinology, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism

المصدر: Genetics in medicine, 24(7), 1425-1436. Lippincott Williams and Wilkins

مصطلحات موضوعية: Adult, Carbon Monoxide, Diffusing capacity of the lung for carbon monoxide, Niemann-Pick type A/B, Niemann-Pick type B, Organomegaly, Recombinant human acid sphingo-myelinase, Niemann-Pick Disease, Type A, Recombinant Proteins, Sphingomyelin Phosphodiesterase, Double-Blind Method, Splenomegaly, Humans, Enzyme Replacement Therapy, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d8858e3c2face69ffd5efd4e9c4736a6Test
https://doi.org/10.1016/j.gim.2022.03.021Test

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PP152 The Assessment Of The Price Of A Medicine: The Possible Application Of Cost-Based Pricing Methods

المؤلفون: Sibren Van Den Berg, Marcel Canoy, Lonneke Timmers, Carla Hollak

المصدر: International Journal of Technology Assessment in Health Care. 38:S89-S90

مصطلحات موضوعية: Health Policy

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::7bb8ea7a408ddd343eecf6f1186f1201Test
https://doi.org/10.1017/s0266462322002641Test

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Lipidoses: The Sphingolipidoses, Lysosomal Acid Lipase Deficiency, and Niemann-Pick Type C

المؤلفون: Carla Hollak

المصدر: Physician's Guide to the Diagnosis, Treatment, and Follow-Up of Inherited Metabolic Diseases ISBN: 9783030677268

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::1c3303bc675e61a867f30f43f9e9fa68Test
https://doi.org/10.1007/978-3-030-67727-5_60Test

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eP280: Continued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial

المصدر: Genetics in Medicine. 24:S176-S177

مصطلحات موضوعية: Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::92369e8a32ff312e301f9907d45361feTest
https://doi.org/10.1016/j.gim.2022.01.315Test

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Two-year results of the ASCEND trial of olipudase alfa adults with chronic acid sphingomyelinase deficiency show parallel improvements in former placebo patients and further improvement in continuing olipudase alfa patients

المصدر: Molecular Genetics and Metabolism. 135:S126-S127

مصطلحات موضوعية: Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::5276339354d6b63808d0ce86f8534cb5Test
https://doi.org/10.1016/j.ymgme.2021.11.337Test

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Time to access to novel anticancer drugs in Europe, a case study in seven European countries

المؤلفون: Colinda Post, Hanneke W.M. Van Laarhoven, Carla Hollak

المصدر: Journal of Clinical Oncology. 40:1586-1586

مصطلحات موضوعية: Cancer Research, Oncology

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::34477250612cebc80f4c1c8ea78786c9Test
https://doi.org/10.1200/jco.2022.40.16_suppl.1586Test

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Editorial

المؤلفون: Robin Lachmann, Carla Hollak

المساهمون: Endocrinology, Amsterdam Gastroenterology Endocrinology Metabolism, AGEM - Inborn errors of metabolism

المصدر: Journal of inherited metabolic disease, 41(5), 899-900. Springer Netherlands

مصطلحات موضوعية: Genetics, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::26ea12704d278023b97e37a407898275Test
https://doi.org/10.1007/s10545-018-0237-5Test

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