Hollak CEM - Search Results

1

Social cognition, emotion regulation and social competence in classical galactosemia patients without intellectual disability.

Hermans ME, Geurtsen GJ, Hollak CEM, Janssen MCH, Langendonk JG, Merckelbach VLV, Oussoren E, Oostrom KJ, Bosch AM. Hermans ME, et al. Among authors: hollak cem. Acta Neuropsychiatr. 2024 Jan 5:1-12. doi: 10.1017/neu.2023.61. Online ahead of print. Acta Neuropsychiatr. 2024. PMID: 38178723

2

[Adults with an inherited metabolic disorder: a rapidly growing population with unique challenges].

Werkgroep INVEST. Werkgroep INVEST. Ned Tijdschr Geneeskd. 2014;158:A7745. Ned Tijdschr Geneeskd. 2014. PMID: 25096043 Review. Dutch.

3

Application of four pricing models for orphan medicines: a case study for lumasiran.

Rosenberg N, Manders E, van den Berg S, Deesker LJ, Garrelfs SF, de Visser SJ, Groothoff JW, Hollak CEM. Rosenberg N, et al. Among authors: hollak cem. Orphanet J Rare Dis. 2024 Dec 23;19(1):485. doi: 10.1186/s13023-024-03446-w. Orphanet J Rare Dis. 2024. PMID: 39716306 Free PMC article.

4

The clinical and biochemical effectiveness and safety of cholic acid treatment for bile acid synthesis defects: a systematic review.

Polak Y, van Dussen L, Kemper EM, Vaz FM, Klouwer FCC, Engelen M, Hollak CEM. Polak Y, et al. Among authors: hollak cem. Orphanet J Rare Dis. 2024 Dec 19;19(1):466. doi: 10.1186/s13023-024-03449-7. Orphanet J Rare Dis. 2024. PMID: 39702264 Free PMC article. Review.

5

Challenges in the identification and quantification of an unknown impurity in chenodeoxycholic acid drug substance.

Bouwhuis N, Polak Y, Schimmel AM, Bijleveld YA, Giera MA, Heijink M, Vaz FM, Bootsma AH, Berg-Lammers LAT, Swart NEL, Hollak CEM, Jacobs BAW, Kemper EM. Bouwhuis N, et al. Among authors: hollak cem. Eur J Pharm Sci. 2024 Dec 4;205:106979. doi: 10.1016/j.ejps.2024.106979. Online ahead of print. Eur J Pharm Sci. 2024. PMID: 39643128 Free article.

6

Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.

Stolwijk NN, Häberle J, Huidekoper HH, Wagenmakers MAEM, Hollak CEM, Bosch AM; E‐IMD and MetabERN Working Group on the Real‐World Use of products for UCD Management. Stolwijk NN, et al. Among authors: hollak cem. J Inherit Metab Dis. 2025 Jan;48(1):e12815. doi: 10.1002/jimd.12815. Epub 2024 Dec 3. J Inherit Metab Dis. 2025. PMID: 39625308 Free PMC article.

7

Stimulating development of innovative medicines in the European Union: does a new definition for unmet medical need add value?

Bloem LT, Leufkens HGM, Berends SE, Vreman RA, Hollak CEM, van Weely S, de Lannoy LM, Bertens PJA, Pasmooij AMG. Bloem LT, et al. Among authors: hollak cem. Drug Discov Today. 2024 Nov 26;30(1):104251. doi: 10.1016/j.drudis.2024.104251. Online ahead of print. Drug Discov Today. 2024. PMID: 39608486 Free article. Review.

8

Acid sphingomyelinase deficiency and Gaucher disease in adults: Similarities and differences in two macrophage storage disorders.

Eskes ECB, van Dussen L, Aerts JMFG, van der Lienden MJC, Maas M, Akkerman EM, van Kuilenburg ABP, Sjouke B, Hollak CEM. Eskes ECB, et al. Among authors: hollak cem. JIMD Rep. 2024 Jul 4;65(5):330-340. doi: 10.1002/jmd2.12420. eCollection 2024 Sep. JIMD Rep. 2024. PMID: 39544689 Free PMC article.

9

Drug pricing models, no 'one-size-fits-all' approach: a systematic review and critical evaluation of pricing models in an evolving pharmaceutical landscape.

Manders EA, van den Berg S, de Visser SJ, Hollak CEM. Manders EA, et al. Among authors: hollak cem. Eur J Health Econ. 2024 Nov 4. doi: 10.1007/s10198-024-01731-w. Online ahead of print. Eur J Health Econ. 2024. PMID: 39495345

10

Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey.

Corazolla EM, Eskes ECB, Veldwijk J, Brands MMMG, Dekker H, van de Mheen E, Langeveld M, Hollak CEM, Sjouke B. Corazolla EM, et al. Among authors: hollak cem. Orphanet J Rare Dis. 2024 Oct 3;19(1):367. doi: 10.1186/s13023-024-03371-y. Orphanet J Rare Dis. 2024. PMID: 39363355 Free PMC article.

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