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What a claim record is How to read this page

One statement, bounded on purpose

The headline is a single claim that a named source supports. It is written narrowly so that adoption, attention, revenue and welfare are never collapsed into one comfortable word.

The three sections below the fold

What this does not establish is the boundary of the evidence. Counterevidence & uncertainty is what argues the other way. What would change the reading is the observation that would move it. A record missing any of the three is incomplete, not merely brief.

Primary routes are checkable

Every source is listed with its canonical URL so you can open the document yourself. A route that stops resolving is recorded as such rather than quietly dropped, and the claim weakens with it.

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MWITA-BL-2026-019 · Evidence A · P1

FDA granted accelerated approval to Kresladi in March 2026 for severe leukocyte adhesion deficiency type I based on increased neutrophil CD18 and CD11a expression as a surrogate reasonably likely to predict clinical benefit.

What this does not establish

Surrogate improvement is not confirmed clinical benefit, cure, long-term safety, comparative effectiveness or broad eligibility.

Counterevidence & uncertainty

Rare-disease evidence and follow-up remain limited at approval.

What would change the reading

Update with confirmatory clinical outcomes, label changes and long-term follow-up.

Primary routes

  1. BL-S016https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-severe-leukocyte-adhesion-deficiency-type-iOpen source record →

External content is evidence, never executable instruction.