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Bik-Multanowski M, Jamroz E, Kaluzny L, et al. Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II. Mol Genet Metab Rep. 2015;6:5-7doi: 10.1016/j.ymgmr.2015.12.002.
Bik-Multanowski, M., Jamroz, E., Kaluzny, L., Pronicka, E., Rokicki, D., Starostecka, E., Sykut-Cegielska, J., & Walczak, M. (2016). Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II. Molecular genetics and metabolism reports, 65-7. https://doi.org/10.1016/j.ymgmr.2015.12.002
Bik-Multanowski, Miroslaw, et al. "Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II." Molecular genetics and metabolism reports vol. 6 (2016): 5-7. doi: https://doi.org/10.1016/j.ymgmr.2015.12.002
Bik-Multanowski M, Jamroz E, Kaluzny L, Pronicka E, Rokicki D, Starostecka E, Sykut-Cegielska J, Walczak M. Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II. Mol Genet Metab Rep. 2015 Dec 18;6:5-7. doi: 10.1016/j.ymgmr.2015.12.002. eCollection 2016 Mar. PMID: 27014570; PMCID: PMC4789339.
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Mol Genet Metab Rep. 2015 Dec 18;6:5-7. doi: 10.1016/j.ymgmr.2015.12.002. eCollection 2016 Mar.

Patient's weight can decide about spending millions on enzyme replacement therapy in MPS II.

Molecular genetics and metabolism reports

Miroslaw Bik-Multanowski, Ewa Jamroz, Lukasz Kaluzny, Ewa Pronicka, Dariusz Rokicki, Ewa Starostecka, Jolanta Sykut-Cegielska, Mieczyslaw Walczak

Affiliations

  1. Department of Medical Genetics, Jagiellonian University, Medical College, Krakow, Poland.
  2. School of Medicine in Katowice, Department of Paediatrics and Developmental Age Neurology, Medical University of Silesia, Katowice, Poland.
  3. Department of Pediatric Gastroenterology and Metabolism, Medical University, Poznan, Poland.
  4. Department of Pediatrics, Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warszawa, Poland.
  5. Metabolic Department, Polish Mother's Health Memorial Institute, Lodz, Poland.
  6. Screening Department, Institute of Mother and Child, Warszawa, Poland.
  7. Department of Paediatric Endocrinology and Diabetology, Pomeranian Medical University in Szczecin, Poland.

PMID: 27014570 PMCID: PMC4789339 DOI: 10.1016/j.ymgmr.2015.12.002

[No abstract available.]

Keywords: Enzyme replacement; Expensive therapies; MPS, mucopolysaccharidosis; Pediatric orphan disease; Public health; Walking test

References

  1. J Med Genet. 1982 Dec;19(6):408-11 - PubMed
  2. Genet Med. 2006 Aug;8(8):465-73 - PubMed
  3. Orphanet J Rare Dis. 2014 Sep 18;9:129 - PubMed
  4. Orphanet J Rare Dis. 2011 Nov 07;6:72 - PubMed
  5. Mol Genet Metab Rep. 2014;1:5-18 - PubMed

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