Leukocyte adhesion deficiency type 1 (LAD-I): US-approved treatments and how Gulf families can access them

Reviewed by the Reserve Meds clinical operations team on 17 October 2026.

What is LAD-I?

Leukocyte adhesion deficiency type 1 is an inherited immune disorder. From birth, children develop serious bacterial and fungal infections, often on the skin and the lining of the nose and mouth. An early sign is late separation of the umbilical cord stump, usually at three weeks or later. In childhood, severe gum disease often leads to tooth loss. A hallmark is that infected areas do not form pus.^[1]

It is caused by variants in the ITGB2 gene and is inherited in an autosomal recessive pattern. It is estimated to occur in about 1 in a million people worldwide, and at least 300 cases have been reported.^[1]

LAD-I in the Gulf

Stem cell transplant has long been the main treatment, and Saudi specialists have published their results. King Faisal Specialist Hospital and Research Centre in Riyadh reported 11 children with LAD-I who had a transplant over 19 years, with an overall event-free survival of 91 percent. Most received bone marrow from an HLA-matched related donor; others received unrelated cord blood or a mismatched related donor.^[2]

Kresladi is for children who have no HLA-matched sibling donor. On 17 October 2026 it was not found in the SFDA drugs list or the Abu Dhabi Department of Health drug code list. It is made from the child's own stem cells, so treatment involves a center that can collect the cells and give the infusion.^{[3, 4, 5]}

US FDA-approved medicines for LAD-I

Each medicine below is approved in the US for a specific group of patients. The wording follows the current US label. Your specialist decides whether any of them is right for the patient.

• Kresladi (marnetegragene autotemcel)
  FDA-approved for children with severe LAD-I caused by variants in both copies of the ITGB2 gene, who have no HLA-matched sibling donor for a stem cell transplant. It is made from the child's own blood stem cells, which are collected, corrected and given back by intravenous infusion. Approved under accelerated approval.^{[6, 3]}
  US FDA approval: 26 March 2026. Gulf registers: Saudi Arabia: not found in the SFDA drugs list. Abu Dhabi: not found in the Abu Dhabi DoH drug code list. Checked 17 October 2026. Kuwait, Qatar and Oman: no public register checked.^{[4, 5]}
  Country guides: Kuwait, Qatar, Oman

How families in the Gulf can access these medicines

For Kresladi. These are made for one patient or given through a specialized procedure, so treatment happens at a qualified center. For most Gulf families that means a referral from the treating specialist to a center abroad that can give the treatment. A local hospital can only give it if the manufacturer agrees to supply that hospital and the regulator approves the import. We cannot promise that a manufacturer will agree.

Who does what

• Your treating specialist decides whether a medicine is right for the patient and writes the letter.
• The hospital checks local supply, files any import request, and gives the treatment safely.
• The national regulator decides whether to approve the import of a medicine that is not registered.
• The manufacturer decides where its medicine can be supplied, especially for cell and gene therapies.
• Reserve Meds helps organize records, coordinates US supply through our wholesale partner, shipping and updates to your family. We do not give medical advice.

Documents you will usually need

• A letter from the treating specialist with the diagnosis, past treatments and why this medicine is being considered
• Reports that confirm the diagnosis, such as genetic, pathology or laboratory results, translated into English where needed
• Recent clinic notes and a list of current medicines
• For a hospital import request: the prescription, the hospital's request form and the written consent of the patient or guardian
• Proof of the US FDA approval and the US prescribing information (we can supply these)

We give you a written timeline estimate once we see the hospital file. The regulator, the treatment center and the manufacturer set their own timelines.

What treatment costs

Quote on request. Your total cost depends on the medicine, the dose, shipping, import steps and coordination. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

How Reserve Meds helps

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help your doctor and hospital prepare the file, arrange US supply and temperature-controlled shipping once approval is in place, and keep your family updated. We coordinate access to FDA-approved medicines only. Investigational medicines are only available through a manufacturer's expanded-access program requested by the treating physician; we can explain that route, but we do not supply them.

Sources for the last answer: ^{[2, 3]}

Related

• Severe combined immunodeficiency (SCID)
• Chronic granulomatous disease
• Named-patient access in the Gulf, explained
• Answers to common access questions
• Cross-border disclosures
• اقرأ هذه الصفحة بالعربية

This page is general health information, not medical advice. It does not replace a consultation with your doctor.