SMAD4-BPIFA1 Axis Governs Airway Antiviral Defense in Myhre Syndrome.
Myhre syndrome is a rare autosomal-dominant disorder caused by gain-of-function (GOF) variants in SMAD4. Although it is primarily recognized as a fibrotic disease, we and others have identified respiratory infection-associated morbidity as a major clinical burden.
Myhre syndrome is a rare autosomal-dominant disorder caused by gain-of-function (GOF) variants in SMAD4. Although it is primarily recognized as a fibrotic disease, we and others have identified respiratory infection-associated morbidity as a major clinical burden.
What the study was
- Study design
- Journal Article (unspecified)
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- Biomolecules
Why it surfaced
Matched watchlist topic 'Rare diseases with high unmet need'. Study design: Journal Article (unspecified). Score: 8/10 (N:3, R:3, D:1, P:1).
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