Time is running out for kids with a rare disease, parents say, unless the FDA acts
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Gilbert Dryden probably only has enough medication to get him through the end of October, his mother, Madison, figures. Limited time: Save 25% on NBC News subscription Get exclusive reporting, live Q&As and ad-free reading. Seven-month-old Gilbert has a rare genetic condition called Barth syndrome, one that can have dire consequences, like heart failure, extreme muscle weakness and a dramatically reduced life expectancy. Children who die early often don’t see their fifth birthday.
AI Summary
Main Idea: The FDA’s repeated delays in reviewing Stealth BioTherapeutics’ drug elamipretide have left families of children with Barth syndrome fearing they will run out of time and treatment.
Key Points:
- Barth syndrome is a rare genetic disease that can cause heart failure, severe weakness, and early death, often before age 5.
- Gilbert Dryden, a baby with the disease, has been helped by elamipretide, but his family says his supply may soon run out.
- Stealth BioTherapeutics says the FDA asked for a third resubmission of the drug application, which could delay a decision by at least six more months.
- Doctors, parents, and some lawmakers say the drug fills a critical need and that more delays could put children at serious risk.
Impact on the People
Negative Impact
FDA delays and rejection of a rare-disease drug can leave families with no treatment and may raise stress and care costs for patients with similar conditions.
Positive Impact
Faster approval of elamipretide could help a tiny group of children avoid heart failure and hospital stays.
How did each entity affect people?
Rate how each entity in this article affected the American people.
- Primary:Gilbert Drydenperson
- Primary:Stealth BioTherapeuticscompany
Candidate Entities8 candidates · 5 weighted vote min · 70% decision
- Gilbert DrydenpersonPrimaryEvaluated
Child patient at the center of the article and the reason the FDA decision matters.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Stealth BioTherapeuticscompanyPrimaryEvaluated
Drug developer seeking approval for elamipretide and directly affected by the FDA delay.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Food and Drug Administrationgovernment agency or bodyPrimaryAI selected
Central regulator whose approval decision and required resubmission drive the entire article.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Madison DrydenpersonMajorAI selected
Named parent whose family’s situation and comments are a major human focus of the story.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Department of Health and Human Servicesgovernment agency or bodyContextCandidate
Oversight department whose spokesman comments on the FDA’s handling of the application.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Kate McCurdypersonContextCandidate
Co-founder of the Barth Syndrome Foundation quoted as a prominent advocate for the drug.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Andrew DrydenpersonMinorCandidate
Gilbert Dryden’s father, mentioned as part of the family affected by the delay.
Include 0 (0%)Ignore 0 (0%)5 to threshold
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