These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

190 related articles for article (PubMed ID: 33980287)

  • 1. Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2).
    Gissen P; Specchio N; Olaye A; Jain M; Butt T; Ghosh W; Ruban-Fell B; Griffiths A; Camp C; Sisic Z; Schwering C; Wibbeler E; Trivisano M; Lee L; Nickel M; Mortensen A; Schulz A
    Orphanet J Rare Dis; 2021 May; 16(1):217. PubMed ID: 33980287
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study.
    Schulz A; Specchio N; de Los Reyes E; Gissen P; Nickel M; Trivisano M; Aylward SC; Chakrapani A; Schwering C; Wibbeler E; Westermann LM; Ballon DJ; Dyke JP; Cherukuri A; Bondade S; Slasor P; Cohen Pfeffer J
    Lancet Neurol; 2024 Jan; 23(1):60-70. PubMed ID: 38101904
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data.
    Specchio N; Gissen P; de Los Reyes E; Olaye A; Camp C; Curteis T; Griffiths A; Butt T; Cohen-Pfeffer J; Slasor P; Sisic Z; Jain M; Schulz A
    PLoS One; 2024; 19(5):e0302382. PubMed ID: 38776275
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Presymptomatic treatment of classic late-infantile neuronal ceroid lipofuscinosis with cerliponase alfa.
    Schaefers J; van der Giessen LJ; Klees C; Jacobs EH; Sieverdink S; Dremmen MHG; Spoor JKH; van der Ploeg AT; van den Hout JMP; Huidekoper HH
    Orphanet J Rare Dis; 2021 May; 16(1):221. PubMed ID: 33990214
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2).
    Gutić M; Milosavljević MN; Safiye T; Milidrag A; Jankovic SM
    Expert Rev Pharmacoecon Outcomes Res; 2023 Jun; 23(5):561-570. PubMed ID: 36994735
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Cerliponase alfa changes the natural history of children with neuronal ceroid lipofuscinosis type 2: The first French cohort.
    Estublier B; Cano A; Hoebeke C; Pichard S; Scavarda D; Desguerre I; Auvin S; Chabrol B
    Eur J Paediatr Neurol; 2021 Jan; 30():17-21. PubMed ID: 33348105
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Intraventricular Cerliponase Alfa Treatment in a Patient with Advanced Neuronal Ceroid Lipofuscinosis Type 2.
    Nakashima S; Hamada M; Kimura T; Tanifuji S; Takahashi A; Yashita D; Kakimoto Y; Matsukawa T; Ishiura H; Toda T
    Intern Med; 2024 Jun; 63(12):1807-1812. PubMed ID: 37926545
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Impact of the COVID-19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatment.
    Mortensen A; Raebel EM; Wiseman S
    Orphanet J Rare Dis; 2022 Jan; 17(1):19. PubMed ID: 35045884
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease: A Retrospective Case Series.
    Wibbeler E; Wang R; Reyes EL; Specchio N; Gissen P; Guelbert N; Nickel M; Schwering C; Lehwald L; Trivisano M; Lee L; Amato G; Cohen-Pfeffer J; Shediac R; Leal-Pardinas F; Schulz A
    J Child Neurol; 2021 May; 36(6):468-474. PubMed ID: 33356800
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Study of Intraventricular Cerliponase Alfa for CLN2 Disease.
    Schulz A; Ajayi T; Specchio N; de Los Reyes E; Gissen P; Ballon D; Dyke JP; Cahan H; Slasor P; Jacoby D; Kohlschütter A;
    N Engl J Med; 2018 May; 378(20):1898-1907. PubMed ID: 29688815
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US Clinics.
    de Los Reyes E; Lehwald L; Augustine EF; Berry-Kravis E; Butler K; Cormier N; Demarest S; Lu S; Madden J; Olaya J; See S; Vierhile A; Wheless JW; Yang A; Cohen-Pfeffer J; Chu D; Leal-Pardinas F; Wang RY
    Pediatr Neurol; 2020 Sep; 110():64-70. PubMed ID: 32684372
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2.
    Lewis G; Morrill AM; Conway-Allen SL; Kim B
    J Child Neurol; 2020 Apr; 35(5):348-353. PubMed ID: 31884868
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Evolution of the retinal function by flash-ERG in one child suffering from neuronal ceroid lipofuscinosis CLN2 treated with cerliponase alpha: case report.
    Rigaudière F; Nasser H; Pichard-Oumlil S; Delouvrier E; Lopez-Hernandez E; Milani P; Auvin S; Delanoë C
    Doc Ophthalmol; 2021 Aug; 143(1):99-106. PubMed ID: 33956290
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease.
    Hammon K; de Hart G; Vuillemenot BR; Kennedy D; Musson D; O'Neill CA; Katz ML; Henshaw JW
    Clin Transl Sci; 2021 Sep; 14(5):1810-1821. PubMed ID: 34076336
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Measurement of Health-Related Quality of Life in Individuals With Rare Diseases in China: Nation-Wide Online Survey.
    Xu RH; Ng SSM; Luo N; Dong D; Zhang S
    JMIR Public Health Surveill; 2023 Oct; 9():e50147. PubMed ID: 37906229
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Ceroid lipofuscinosis type 2 disease: Effective presymptomatic therapy-Oldest case of a presymptomatic enzyme therapy.
    Breuillard D; Ouss L; Le Normand MT; Denis TS; Barnerias C; Robert MP; Eisermann M; Boddaert N; Caillaud C; Bahi-Buisson N; Desguerre I; Aubart M
    Eur J Neurol; 2024 Sep; 31(9):e16324. PubMed ID: 38693756
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease).
    Dulz S; Schwering C; Wildner J; Spartalis C; Schuettauf F; Bartsch U; Wibbeler E; Nickel M; Spitzer MS; Atiskova Y; Schulz A
    Br J Ophthalmol; 2023 Oct; 107(10):1478-1483. PubMed ID: 35772852
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy.
    Specchio N; Pietrafusa N; Trivisano M
    Ther Clin Risk Manag; 2020; 16():213-222. PubMed ID: 32280231
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Cerliponase Alfa: First Global Approval.
    Markham A
    Drugs; 2017 Jul; 77(11):1247-1249. PubMed ID: 28589525
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa.
    Guelbert N; Espitia Segura OM; Amoretti C; Arteaga Arteaga A; Atanacio NG; Bazan Natacha S; Carvalho EDF; Carvalho de Andrade MDF; Denzler IM; Durand C; Ribeiro E; Giugni JC; González G; González Moron D; Guelbert G; Hernández Rodriguez ZJ; Embiruçu Emilia K; Kauffman MA; Mancilla NI; Marcon L; Marques Pereira A; Fischinger Moura de Souza C; Muñoz VA; Naranjo Flórez RA; Pessoa AL; Ruiz MV; Solano Villareal ML; Spécola N; Tavera LM; Tello J; Troncoso Schifferli M; Ugrina S; Vaccarezza MM; Vergara D; Villanueva MM
    Mol Genet Metab Rep; 2024 Mar; 38():101060. PubMed ID: 38469103
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 10.