The Role of Pharmacological Chaperones in Fabry Disease Treatment
Pharmacological chaperones have emerged as a promising treatment modality for Fabry disease. Unlike enzyme replacement therapies, chaperones work by stabilizing the faulty enzyme to enhance its function naturally.
Migalastat, the first approved pharmacological chaperone, is effective for patients with specific genetic mutations. It offers the advantage of oral administration, improving patient compliance.
The Fabry Disease Market is expanding as more patients seek alternatives to intravenous ERTs. The development of chaperones opens new avenues for personalized medicine in rare diseases.
The challenge remains in identifying suitable candidates for chaperone therapy through genetic testing, which is becoming more accessible.
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