Latest Advances

  • Condition: Fabry Disease (FD)
  • Journal: International journal of molecular sciences
  • Treatment Used: Enzyme Replacement Therapy (Agalsidase Alfa and Agalsidase Beta; ERT) and Migalastat
  • Number of Patients: 0
  • Published —
This article provides an overview of currently approved and emerging new therapies for adult patients with Fabry disease (genetic enzyme disease; FD).
  • Condition: Fabry Disease
  • Journal: International journal of molecular sciences
  • Treatment Used: Enzyme Replacement Therapy
  • Number of Patients: 0
  • Published —
The study researched Fabry disease.
  • Condition: Diffuse Hyperkeratotic Papules of the Lower Abdomen and Genital Region
  • Journal: Der Internist
  • Treatment Used: Enzyme Replacement Therapy or Chaperone Therapy
  • Number of Patients: 1
  • Published —
This case report describes a patient with diffuse hyperkeratotic papules of the lower abdomen and genital region that was treated using enzyme replacement therapy or chaperone therapy.
  • Condition: Cerebral Progression in Fabry Disease (FD)
  • Journal: Journal of neurology, neurosurgery, and psychiatry
  • Treatment Used: Enzyme Replacement Therapy (ERT)
  • Number of Patients: 149
  • Published —
This study determined the effect of enzyme replacement therapy (ERT) and progression of white matter lesions (WMLs) in patients with Fabry disease (genetic enzyme disorder; FD).
  • Condition: Fabry Disease
  • Journal: Advances in therapy
  • Treatment Used: Enzyme replacement therapy
  • Number of Patients: 367
  • Published —
The study discusses the symptoms and quality of life in patients with Fabry disease.
  • Condition: Stroke in Fabry's disease
  • Journal: Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia
  • Treatment Used: Enzyme replacement therapy (ERT)
  • Number of Patients: 7513
  • Published —
The aim of the study was to determine the benefit of enzyme replacement therapy (ERT) for stroke prevention in Fabry's patients.
  • Condition: Male Patients with Fabry Disease
  • Journal: Genetics in medicine : official journal of the American College of Medical Genetics
  • Treatment Used: Migalastat
  • Number of Patients: 14
  • Published —
This study evaluated the outcomes in patients with Fabry disease (genetic enzyme disorder) receiving migalastat by phenotype.
  • Condition: Fabry Disease
  • Journal: Clinical pharmacology and therapeutics
  • Treatment Used: Oral Chaperone Therapy Migalastat
  • Number of Patients: 14
  • Published —
This study evaluated migalastat effectiveness and biomarker changes in patients with Fabry disease (genetic disorder).
  • Condition: Female patients with Fabry disease
  • Journal: Molecular genetics and metabolism
  • Treatment Used: Enzyme replacement therapy
  • Number of Patients: 0
  • Published —
The study researched the effects of enzyme replacement therapy for female patients with Fabry disease.
  • Condition: Fabry Disease
  • Journal: Molecular genetics and metabolism
  • Treatment Used: Enzyme Replacement Therapy
  • Number of Patients: 0
  • Published —
This review of the literature analyzed the effectiveness of enzyme replacement therapy (ERT) to treat pediatric patients with Fabry Disease (rare genetic disease with a deficiency of an enzyme called alpha-galactosidase A).
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