Results 101 to 110 of about 550 (133)

Evaluation of the frequency and characteristics of drug hypersensitivity reactions in hospitalized children: Real life-cohort study. [PDF]

World Allergy Organ J
Büyük Yaytokgil Ş, Selmanoglu A, Kulhas Celik I, Şengül Emeksiz Z, Ginis T, Karaatmaca B, Toyran M, Civelek E, Dibek Misirlioğlu E.   +8 more
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Unlocking access: a comprehensive analysis of medicines accessibility for rare diseases in Thailand. [PDF]

Orphanet J Rare Dis
Suwattanapreeda S, Hirunrassamee S, Sooksriwong C, Maluangnon K, Chuachantra T, Kuchaisit K, Osirisakul N.   +6 more
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Products with Black Box Warnings [PDF]

, 2006
Woelfel, Joseph A.
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The impact of rare diseases on the quality of life in paediatric patients: current status. [PDF]

Front Public Health
Dumbuya JS, Zeng C, Deng L, Li Y, Chen X, Ahmad B, Lu J.   +6 more
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Clinical data required for the approval of pediatric pharmaceuticals in Japan. [PDF]

BMC Pediatr
Haigo H, Matsuda K, Shikano M.
europepmc   +1 more source

Recombinant human alpha-N-acetylglucosamine-6-sulfatase delivered to Sanfilippo D mice with repeated intracerebroventricular injections corrects CNS pathology. [PDF]

PLoS One
Austin GL, Wang F, Le SQ, Sorensen A, Li S, Foong LC, Singamsetty S, Wood J, Chou TF, Dickson PI.   +9 more
europepmc   +1 more source

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Safety and efficacy of enzyme replacement therapy with idursulfase beta in children aged younger than 6years with Hunter syndrome

Molecular Genetics and Metabolism, 2015
Idursulfase beta (Hunterase®) has been used for enzyme replacement therapy (ERT) of patients with mucopolysaccharidosis II (MPS II, Hunter syndrome) aged 6 years or older since 2012 in Korea. The objective of this study was to evaluate the safety and efficacy of ERT with idursulfase beta in Hunter syndrome children younger than 6 years.
Young Bae, Sohn, Sung Yoon, Cho, Jieun, Lee, Yonghee, Kwun, Rimm, Huh, Dong-Kyu, Jin   +5 more
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The efficacy of intracerebroventricular idursulfase‐beta enzyme replacement therapy in mucopolysaccharidosis II murine model: heparan sulfate in cerebrospinal fluid as a clinical biomarker of neuropathology

Journal of Inherited Metabolic Disease, 2018
AbstractMucopolysaccharidosis II (MPS II) is caused by a deficiency of iduronate‐2‐sulfatase that results in accumulation of glycosaminoglycans (GAG), including heparan sulfate (HS), which is considered to contribute to neuropathology. We examined the efficacy of intracerebroventricular (ICV) enzyme replacement therapy (ERT) of idursulfase‐beta (IDS‐β)
Young Bae, Sohn, Ah-Ra, Ko, Mi-Ran, Seong, Soyeon, Lee, Mi Ra, Kim, Sung Yoon, Cho, Jung-Sun, Kim, Makoto, Sakaguchi, Takahiro, Nakazawa, Motomichi, Kosuga, Joo Hyun, Seo, Torayuki, Okuyama, Dong-Kyu, Jin   +12 more
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Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: a phase 3, two-part study compared to a historical placebo cohort.

Genetics in medicine : official journal of the American College of Medical Genetics
To investigate the efficacy and safety of idursulfase beta (0.5 mg/kg weekly) in the treatment of mucopolysaccharidosis II (MPS II), compared with a historical placebo from a previous idursulfase trial (TKT024).The study comprised two sequential parts. In Part 1, a randomized, double-blind study, idursulfase or idursulfase beta were given for 52 weeks.
Young Bae, Sohn, Aram, Yang, Min-Sun, Kim, Jinsup, Kim, Jae Seong, Kim, Youngsin, Oh, Dong-Kyu, Jin   +6 more
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