The Fabrazyme shortage--a call to action for metabolic physicians.
The recent shortages of enzyme replacement therapy for Fabry disease have highlighted areas of vulnerability for patients who require this treatment. Guidelines on allocation of limited stock of enzyme replacement therapy are of use for clinicians dealing with the current shortages. However, the community of metabolic physicians must advocate for changes that will minimize the impact of future drug shortages for their patients with lysosomal storage diseases.
Adult Metabolic Diseases Clinic, Level 4—2775 Laurel Street, Vancouver, BC, V5Z 1M9, Canada. Sandra.Sirrs@vch.ca
Sandra
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Sirrs
D056947:Enzyme Replacement Therapy; D006801:Humans; D007527:Isoenzymes / Q000008:administration & dosage / Q000600:supply & distribution*; D017410:Practice Guidelines as Topic; D000519:alpha-Galactosidase / Q000008:administration & dosage / Q000600:supply & distribution*
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta