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Mucopolysaccharidosis type I (MPS I, Hurler syndrome): US-approved treatments and how Gulf families can access them

Creator: Reserve Meds
Published: 2026-10-17

A plain guide for families in the Gulf, checked against US FDA labels, Gulf drug registers and published medical research.

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

MPS I (Hurler syndrome): the short answer

The US FDA has approved one medicine on this page for MPS I (Hurler syndrome), each for specific patients. One of them is on at least one Gulf register we checked, so ask your hospital about local supply first. Otherwise the hospital can request a named-patient import. Reserve Meds is a digital health company. We do not diagnose or prescribe.

US FDA-approved medicines on this page: Aldurazyme.
Gulf registration (checked 17 October 2026): see each medicine below.
Legal route: Local supply where listed; otherwise a named-patient import request filed by the treating hospital.
Price: Quote on request.

What is MPS I (Hurler syndrome)?

Mucopolysaccharidosis type I (MPS I) is an inherited condition that affects many parts of the body. It was once divided into Hurler, Hurler-Scheie and Scheie syndromes, from most to least severe. Today it is described as severe or attenuated MPS I.^[1]

Children with severe MPS I usually show signs in the first year of life. Features can include a large head, coarse facial features, an enlarged liver and spleen, heart valve problems, a large tongue, clouding of the cornea, hearing loss, joint stiffness, short stature and skeletal changes. Narrow airways can cause frequent infections and sleep apnea.^[1]

MPS I is caused by missing or low activity of the enzyme alpha-L-iduronidase. Early treatment with stem cell transplantation and/or enzyme replacement therapy is linked with better outcomes, but diagnosis is often delayed because early symptoms are not specific.^[2]

MPS I (Hurler syndrome) in the Gulf

In a pilot study at Farwaniya Hospital in Kuwait, 618 newborns were screened for MPS I over 12 months as a step toward national newborn screening. The authors note that the incidence of MPS I in Kuwait is unknown and that late diagnosis is common.^[2]

US FDA-approved medicines for MPS I (Hurler syndrome)

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

Aldurazyme (laronidase)
FDA-approved for adults and children with the Hurler and Hurler-Scheie forms of MPS I, and patients with the Scheie form who have moderate to severe symptoms. It is an enzyme replacement therapy given by intravenous infusion.^[3]
Gulf registers: Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register.^{[4, 5]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt

How families in the Gulf can access these medicines

If the medicine is listed in your country. Ask your specialist and the hospital pharmacy about local supply first. A listing does not mean every hospital stocks the medicine, and another brand of the same medicine may also be registered, but local supply is usually the fastest route and you may not need us.

If it is not registered. The usual legal route is a named-patient request: the treating hospital, on your specialist's written request, asks the national regulator for permission to import the medicine for one patient, using the US FDA approval as the reference. In Saudi Arabia the SFDA Special Access Program guidance covers these requests; in Bahrain, NHRA Circular 41 of 2020 sets the procedure. Other Gulf countries have their own procedures, which the hospital pharmacy confirms.^{[6, 7]} Once approval is in place, Reserve Meds arranges supply through a US-licensed pharmaceutical wholesale partner to the hospital pharmacy.

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 the medicine is being considered
Reports that confirm the diagnosis, such as laboratory, genetic, imaging or pathology 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.

Ask us about treatment for MPS I (Hurler syndrome)

Tell us the medicine, your country and a WhatsApp or phone number. We reply within one business day. On WhatsApp we usually reply the same day.

Frequently asked questions

Is there an FDA-approved treatment for MPS I (Hurler syndrome)?

Yes. The medicine on this page has US FDA approval for specific groups of patients: Aldurazyme. The exact conditions are in each US label and are summarized above. Your specialist decides whether any of them fits.

Is Aldurazyme available in Saudi Arabia or the UAE?

Saudi Arabia: listed in the SFDA drugs list. Abu Dhabi: has an active code in the Abu Dhabi DoH drug code list. Checked 17 October 2026. UAE federal status, Kuwait, Qatar, Bahrain and Oman: not verified in a public register. A listing in Abu Dhabi does not show federal UAE status.

How can a family in the Gulf get Aldurazyme?

Ask the treating hospital about local supply first. If it is not available, the hospital can file a named-patient import request with the national regulator on your specialist's written request. Once approved, we arrange US supply to the hospital pharmacy.

How much does treatment cost?

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

Is enzyme replacement the only option?

No. Researchers in Kuwait note that stem cell transplantation and/or enzyme replacement therapy started early are linked with better outcomes. The choice depends on the child's age and form of MPS I and is made by a specialist center.

How is MPS I confirmed?

By measuring the activity of the alpha-L-iduronidase enzyme and by genetic testing of the IDUA gene, as described in the Kuwaiti screening study.

Sources for the last two answers: ^[2] ^[2]

Sources

Checked on 17 October 2026. Labels, registers and regulator rules change. Your hospital pharmacy confirms the current position before filing.

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