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Childhood hypophosphatasia: to treat or not to treat.
Rush ET. Rush ET. Orphanet J Rare Dis. 2018 Jul 16;13(1):116. doi: 10.1186/s13023-018-0866-7. Orphanet J Rare Dis. 2018. PMID: 30012160 Free PMC article. Review.
The disease is life-threatening when manifesting within the first six months of life, excepting the extremely rare benign perinatal hypophosphatasia. Childhood hypophosphatasia, defined as onset of symptoms between six months and eighteen years, can manifest as rick …
The disease is life-threatening when manifesting within the first six months of life, excepting the extremely rare benign perinatal hypophos …
Treatment of childhood hypophosphatasia with nonsteroidal antiinflammatory drugs.
Girschick HJ, Seyberth HW, Huppertz HI. Girschick HJ, et al. Bone. 1999 Nov;25(5):603-7. doi: 10.1016/s8756-3282(99)00203-3. Bone. 1999. PMID: 10574582
The benefit of this treatment was evaluated clinically and by measurement of renally (PGE2) and systemically (PGE-M) derived prostaglandins (PG) in urine before and during therapy. After treatment with NSAID all six patients showed marked clinical improvement with reduced …
The benefit of this treatment was evaluated clinically and by measurement of renally (PGE2) and systemically (PGE-M) derived prostaglandins …
Asfotase alfa treatment for 1 year in a 16 year-old male with severe childhood hypophosphatasia.
Bowden SA, Adler BH. Bowden SA, et al. Osteoporos Int. 2018 Feb;29(2):511-515. doi: 10.1007/s00198-017-4267-x. Epub 2017 Oct 18. Osteoporos Int. 2018. PMID: 29046930
We describe the clinical outcome of asfotase alfa therapy in a 16-year-old boy with severe childhood hypophosphatasia (HPP), who began therapy at age 15 years. ...He was started on asfotase alfa 2 mg/kg given subcutaneously thrice weekly. He had marked cli
We describe the clinical outcome of asfotase alfa therapy in a 16-year-old boy with severe childhood hypophosphatasia ( …
Effective NSAID treatment indicates that hyperprostaglandinism is affecting the clinical severity of childhood hypophosphatasia.
Girschick HJ, Schneider P, Haubitz I, Hiort O, Collmann H, Beer M, Shin YS, Seyberth HW. Girschick HJ, et al. Orphanet J Rare Dis. 2006 Jun 28;1:24. doi: 10.1186/1750-1172-1-24. Orphanet J Rare Dis. 2006. PMID: 16803637 Free PMC article.
METHODS: We analyzed the changes in clinical symptoms and the prostaglandin (PG) metabolism in response to treatment with non-steroidal anti-inflammatory drugs (NSAIDs) in six children affected by childhood HP. ...Consequently, NSAID treatment does improve the clinical
METHODS: We analyzed the changes in clinical symptoms and the prostaglandin (PG) metabolism in response to treatment with non-steroid …