USANew treatments and approvals
Marnetegragene autotemcel receives FDA approval for severe LAD-I immunodeficiency
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The FDA has approved marnetegragene autotemcel for severe leukocyte adhesion deficiency type I. In this disease, white blood cells do not work properly. It causes life-threatening, recurrent infections in children. In the treatment, the patient's own stem cells are genetically corrected. Nine patients were treated in the clinical study. In all of them, the immune system was reported to have improved lastingly.
What does it mean for patients and families?
An additional option to stem cell transplantation has become available for patients without a suitable related donor. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Leukocyte adhesion deficiency type I (LAD-I)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.