The metabolic landscape has evolved from the use of diet management to the introduction of enzyme replacement therapies and gene therapy developments on the horizon for some indications. Current metabolic dynamics the REACH team is tracking include:
- The development of disease-altering AAV or mRNA gene therapies for genetic diseases like Glycogen Storage Disorder (GSD) 1a and Phenylketonuria
- Novel delivery mechanisms of enzyme replacement therapies such as subcutaneous administration
- Payer coverage dynamics associated with diet therapies and subsequent out-of-pocket costs
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