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Carla E. M. Hollak

Montefiore Medical Center

78H-index
432Paper Count
1.9WCitation Count
Published Papers 199
Publication Date
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
err2026-04-28
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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
err2026-04-27
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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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Fast and controlled access to tofersen for SOD1-related amyotrophic lateral sclerosis in The Netherlands; experiences using the orphan drug access protocol
err2026-04-13
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PREAI
errSean W. Willemse; Carla E.M. Hollak; Niels D. Reijnhout; Carelle C. Reparon-Schuijt; Anke A.M.G. Pisters-van Roy; Koen C. Demaegd; Ruben P.A. van Eijk; Leonard H. van Den Berg; Michael A van Es
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Safety and Effectiveness of Pharmacy Compounded Chenodeoxycholic Acid Capsules for Patients With Cerebrotendinous Xanthomatosis
err2026-03-07
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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?
err2026-02-17
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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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Pharmacokinetic Cross-Over Study of Pharmacy-Compounded Chenodeoxycholic Acid Capsules Compared to Authorized Capsules
err2025-11-27
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errNatalja Bouwhuis; Bart A. W. Jacobs; Soumia Majait; Frédéric M. Vaz; Carla E. M. Hollak; Eleonora L. Swart; E. Marleen Kemper
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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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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
err2025-10-28
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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 Leukodystrophy
err2025-08-02
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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
err2025-08-02
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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
err2025-07-28
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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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Exploring Exhaled Breath Analysis in Adults With Chronic Visceral Acid Sphingomyelinase Deficiency to Identify Potential Biomarkers of Pulmonary Involvement
err2025-07-27
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errEline C. B. Eskes; Bauke V. Schomakers; Michel van Weeghel; Suzanne W. J. Terheggen-Lagro; Lilian J. Meijboom; Carla E. M. Hollak; Paul Brinkman; Barbara Sjouke
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Editorial Comment to Regulatory News
err2025-07-23
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PREAI
errCarla E. M. Hollak; Natalja Bouwhuis
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Effectiveness and Safety of Personalized Cholic Acid Treatment in Patients With Bile Acid Synthesis Defects
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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The value of MR spectroscopy and MR elastography in assessing hepatic involvement of chronic visceral acid sphingomyelinase deficiency in adults
err2025-06-01
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PREAI
errEskes, Eline C. B.; van den Berg-Faaij, Sandra A. M.; Wassenaar, Nienke P. M.; Hollak, Carla E. M.; Nederveen, Aart J.; Sjouke, Barbara
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New opportunities for accessing promising non-oncological orphan drugs
err2025-05-01
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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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Product Development of High-Dose Ambroxol HCl Capsules for an n-of-1 Clinical Trial Involving Dutch Patients with Gaucher Disease Type 3
err2025-03-25
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errLe, Hoang Lan; Bouwhuis, Natalja; Hollak, Carla E. M.; Wilhelm, Abraham J.; Gerards, Anne-Loes E.; Bijleveld, Yuma A.; Swart, Eleonora L.
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