Results 101 to 110 of about 783 (129)

An international observational study on transition of care from paediatric to adult services for patients with mucopolysaccharidosis II. [PDF]

BMC Health Serv Res
Stepien KM, Ayodele O, Muenzer J, Giugliani R, Jackson C, Meade L, Scarpa M, Lampe C, Audi J, Jain S, Toro MD.   +10 more
europepmc   +1 more source

Production of active human iduronate-2-sulfatase (IDS) enzyme in Nicotiana benthamiana. [PDF]

Sci Rep
Sinha MH, Mehtab T, Entesari M, Nguyen HH, Yun A, Hwang I.   +5 more
europepmc   +1 more source

Perceptions of patients and caregivers toward the management of rare disease in Malaysia: a qualitative research study. [PDF]

Int J Technol Assess Health Care
Supian A, Shafie AA, Ngu LH, Ayob H, Chaiyakunapruk N.   +4 more
europepmc   +1 more source

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond. [PDF]

BioDrugs
Zanetti A, Tomanin R.
europepmc   +1 more source

Analysis of fatal outcomes of patients with mucopolysaccharidosis type II according to the Russian mucopolysaccharidosis registry. [PDF]

World J Clin Pediatr
Buchinskaya N, Vechkasova A, Vashakmadze N, Namazova-Baranova L, Ivanov D, Zakharova E, Kutsev S, Kostik M.   +7 more
europepmc   +1 more source

AMCP Market Insights: The role of managed care in improving patient care in mucopolysaccharidoses. [PDF]

J Manag Care Spec Pharm
Flavin B, Haumschild R, Klein TK, McKay K, Mok T, O'Neill C, Rice M, Stephens K, Stoltz E.   +8 more
europepmc   +1 more source

The imperative for national legislation on rare diseases in China: A policy review and call to action. [PDF]

Intractable Rare Dis Res
Li Y, Liu Z, Huang R, Liu Y.
europepmc   +1 more source

Rarely listed essential medicines in 158 national lists. [PDF]

J Pharm Policy Pract
Ul Haq MZ, Heredia C, Rizvi A, Workentin A, Persaud N.   +4 more
europepmc   +1 more source