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Carla E. M. Hollak

Montefiore Medical Center

78H指数
434论文数
1.9W被引数
收录论文 200
发表时间
Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: ASCEND Trial Final Results成人鞘磷脂酶缺乏症患者经Olipudase Alfa酶替代疗法治疗5年后临床结局持续改善:ASCEND试验最终结果
err2026-04-28
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errOAAI
errWasserstein, Melissa P.; Hollak, Carla E.; Barbato, Antonio; Gallagher, Renata C.; Giugliani, Roberto; Guelbert, Norberto Bernardo; Hennermann, Julia B.; Ikezoe, Takayuki; Lachmann, Robin; Lidove, Olivier; Mabe, Paulina; Mengel, Eugen; Scarpa, Maurizio; Senates, Ebubekir; Tchan, Michel; Thurberg, Beth L.; Villarrubia, Jesus; Armstrong, Nicole M.; Bhushan, Shashi; Kim, Yong; Kumar, Monica
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Effectiveness of Riboflavin in Inherited Metabolic Diseases: A Systematic Review核黄素在遗传代谢病中的有效性:一项系统综述
err2026-04-27
err0
errOAAI
errBregje Jaeger; Nina N. Stolwijk; Femke Aaldering; Charlotte A. Ruys; Marije Smits; Nicole I. Wolf; Carla E. M. Hollak; Annet M. Bosch
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Safety and Effectiveness of Pharmacy Compounded Chenodeoxycholic Acid Capsules for Patients With Cerebrotendinous Xanthomatosis药房配制的鹅去氧胆酸胶囊对 cerebrotendinous xanthomatosis 患者的安全性与有效性
err2026-03-07
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errOAAI
errNatalja Bouwhuis; Soumia Majait; Yasmin Polak; Aylene de Jong; Bart A. W. Jacobs; Frédéric M. Vaz; Willemijn G. Leen; E. Marleen Kemper; Eleonora L. Swart; Carla E. M. Hollak
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Medicine Development and Access for Rare Diseases: Can We Do Better?罕见病药物研发与可及性:我们能做得更好吗?
err2026-02-17
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errOAAI
errHollak, Carla E. M.; Rosenberg, Noa; Post, Colinda; Stolwijk, Nina; Manders, Evert; Schoenmakers, Daphne; Penninx, Bart; Reijnhout, Niels; Verdeyen, Kathelijn; Jonker, Roel; Bosch, Annet M.; Langeveld, Mirjam; van der Wel, Vincent; de Visser, Saco; van den Berg, Sibren
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Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries
err2025-11-01
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errOAAI
errRosenberg, N.; Post, H. C.; Schutte, T.; De Visser, S. J.; Bartelink, I.; Pasmooij, A. M. G.; Van Laarhoven, H. W. M.; Hollak, C. E. M.
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Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix管理准入协议以减少罕见病药物报销挑战的驱动因素:矩阵的开发
err2025-10-28
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errOAAI
errMarcelien H. E. Callenbach; Sibren van den Berg; Alisa Hulsbosch; Carla E. M. Hollak; Christine Leopold; Aukje K. Mantel-Teeuwisse; Wim G. Goettsch
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ARSA Variants Associated With Cognitive Decline and Long-Term Preservation of Motor Function in Metachromatic LeukodystrophyARSA变体与认知能力下降及异染性脑白质营养不良中运动功能的长期维持相关
err2025-08-02
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errOAAI
errShanice Beerepoot; Daphne H. Schoenmakers; Francesca Fumagalli; Samuel Groeschel; Ludger Schöls; Raphael Schiffmann; Sheila Wong; Odile Boespflug-Tanguy; Caroline Sevin; Yann Nadjar; Annette Bley; Fanny Mochel; Morten A. Horn; Cristina Baldoli; Sara Locatelli; Holger Hengel; Lucia Laugwitz; Carla E. M. Hollak; Volkmar Gieselmann; Marjo S. van der Knaap; Nicole I. Wolf
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Effectiveness of Pyridoxal-5′-Phosphate in PNPO Deficiency: A Systematic Review吡哆醛-5′-磷酸在PNPO缺乏症中的有效性:一项系统评价
err2025-08-02
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errOAAI
errNina N. Stolwijk; Laura van Dussen; Niels D. Reijnhout; Marion M. M. G. Brands; Bregje Jaeger; Peter T. Clayton; Carla E. M. Hollak; Annet M. Bosch
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Access to EU-Authorized Nonreimbursed Anticancer Medicines: An Explorative Survey Among Dutch Medical Oncologists获得欧盟授权的非报销抗癌药物:一项针对荷兰医学肿瘤学家的探索性调查
err2025-07-28
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errOAAI
errH. Colinda Post; Charlotte H. C. Bomhof; Maartje Schermer; Carla E. M. Hollak; Hanneke W. M. van Laarhoven; Tim Schutte; Eline M. Bunnik
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Effectiveness and Safety of Personalized Cholic Acid Treatment in Patients With Bile Acid Synthesis Defects个性化胆汁酸治疗在胆汁酸合成缺陷患者中的有效性与安全性
err2025-07-11
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PREAI
errYasmin Polak; Elles Marleen Kemper; Marc Engelen; Femke C. C. Klouwer; Kevin Berendse; Frédéric M. Vaz; Bart G. P. Koot; Eleonora (Noortje) L. Swart; Carla E. M. Hollak
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New opportunities for accessing promising non-oncological orphan drugs获取有前景的非肿瘤学罕见病药物的新机会
err2025-05-01
err0
PREAI
errHollak, Carla E. M.; Reparon-Schuijt, Carelle C.; Verdeyen, Kathelijn; Deesker, Lisa J.; Groothoff, Jaap W.; Vos, Josephine M. I.; Huisman, Atse H.; van der Flier, Silvia; Roy, Anke A. M. G. Pisters-van
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