Journal of Clinical & Experimental Pharmacology

Department of Propedeutics of Internal Diseases, Faculty of Medicine, Medical University, Plovdiv, Bulgaria

Publications

• Case Report   
  VIMIZIN (Elosulfatasealpfa) in an Adult Patient with Mucopolysaccharidosis Type IV Ð
  Author(s): Mariela Geneva-Popova*
  
  Lysosomal diseases include a group of over 40 inherited metabolic diseases due to enzyme deficiency. In them, the accumulation of undegraded substrate in the cells is observed. They are chronic diseases with a heterogeneous clinical picture. Mucopolysaccharidosis type IVA (Morquio syndrome) is a rare form of these diseases and occurs in a frequency of 1: 200,000 patients. Treating this condition is difficult, expensiveÑ€, and often ineffective. We report a patient, a 22-year-old man suffering from Morquio syndrome, who was admitted to the hospital in a serious condition with an inability to move due to musculoskeletal problems (the patient walks 0 meters in a 6-minute walking test) and inability to climb stairs. In 2018, treatment with Vimizin (elosulfatasealfa) was started at a dosage of 2 mg/kg body weight every week. The patient's condition was moni.. View more»
  

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