Who’s going to run the FDA?
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By
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Damian Garde
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July 9, 2026
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Clinical Scorecard: Who Will Lead the FDA?
At a Glance
| Category | Detail |
|---|
| Condition | NGLY1 deficiency |
| Key Mechanisms | Gene therapy targeting genetic mutations |
| Target Population | Children with ultra-rare genetic conditions |
| Care Setting | Regulatory environment for drug approval |
Key Highlights
- Matt Wilsey is advocating for FDA approval of a gene therapy for his daughter with NGLY1 deficiency.
- Ten children have been treated with the gene therapy, and Wilsey believes it is effective.
- The FDA requires extensive data on manufacturing and efficacy, which Wilsey struggles to provide.
Guideline-Based Recommendations
Diagnosis
- Diagnosis of NGLY1 deficiency through genetic testing.
Management
- Consider gene therapy as a potential treatment option.
Monitoring & Follow-up
- Monitor patients for developmental challenges and treatment efficacy.
Risks
- Potential risks associated with gene therapy and regulatory hurdles.
Patient & Prescribing Data
Children diagnosed with NGLY1 deficiency.
Gene therapy is in experimental stages with limited patient data.
Clinical Best Practices
- Engage with regulatory bodies early in the treatment development process.
- Collaborate with experts in gene therapy and rare diseases.
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