Results 91 to 100 of about 550 (133)

Cardiac manifestations in adult patients with inherited metabolic disease: A single-center experience. [PDF]

Mol Genet Metab Rep
Sadiku F, Rutz T, Superti-Furga A, Monney P, Tran C.   +4 more
europepmc   +1 more source

Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan. [PDF]

Orphanet J Rare Dis
Nakamura K, Sakai N, Hossain MA, Eisengart JB, Yamamoto T, Tanizawa K, So S, Schmidt M, Sato Y.   +8 more
europepmc   +1 more source

Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs. [PDF]

Ther Adv Rare Dis
Awada N.
europepmc   +1 more source

Strategy for reproductive and developmental toxicity (DART) studies for marketing applications in pharmaceutical development. [PDF]

Curr Res Toxicol
Kagawa Y, Osawa T, Koyama N, Tanaharu T, Kigami D.   +4 more
europepmc   +1 more source

Früherkennung bei Mukopolysaccharidose Typ II [PDF]

, 2017
Güth, Christian
core   +2 more sources

Risk and clinical characteristics of spinal cord compression across different mucopolysaccharidosis types: A retrospective cohort study. [PDF]

Medicine (Baltimore)
Kim I, Sung J, Ahn YJ, Im M, Kim MJ, Park SJ, Cho SY, Cho SY.   +7 more
europepmc   +1 more source

Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023. [PDF]

Front Pharmacol
Nie R, Zhao Z, Zhang Y, Xu B, Zhang W.
europepmc   +1 more source

Drug-associated hearing impairment in children: a disproportionality analysis of the FDA adverse event reporting system. [PDF]

Front Pharmacol
Liu J, Tan J, Xiao Q, Bai Y, Chang E, Su C, Wei Y, Zhong H, Wei W.   +8 more
europepmc   +1 more source

Drug-Associated Tendinopathies and Ligament Disorders: Results from a Retrospective Pharmacovigilance Study Using Disproportionality Analysis. [PDF]

Hosp Pharm
Sridharan K.
europepmc   +1 more source

Single-cell RNA sequencing reveals important role of monocytes and macrophages during mucopolysaccharidosis treatment. [PDF]

Sci Rep
Zhu G, Xie Y, Li L, Li R, Sun Y, Zhou T, Cun Y.   +6 more
europepmc   +1 more source