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Barth 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.

Barth syndrome: the short answer

For Barth syndrome, the US FDA has approved one medicine covered on this page, for a specific group of patients: Forzinity. Where a medicine is not registered in your country, the treating hospital can ask the national regulator for permission to import it for one named patient. Reserve Meds is a digital health company that helps families and hospitals with that process. We do not diagnose or prescribe.

US FDA-approved medicines on this page: Forzinity.
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 Barth syndrome?

Barth syndrome is a rare condition marked by an enlarged and weakened heart (dilated cardiomyopathy), weak muscles used for movement, recurrent infections due to low numbers of a type of white blood cell (neutropenia), and short stature. It occurs almost only in males. Heart problems often start at birth or in the first months of life and can lead to heart failure. Muscle weakness can delay crawling and walking, and tiredness during effort is common.^[1]

It is caused by changes in the TAFAZZIN gene and is inherited in an X-linked recessive pattern. It is estimated to affect 1 in 300,000 to 400,000 people worldwide.^[1]

Barth syndrome in the Gulf

We did not find published Gulf-specific data on Barth syndrome, so we do not give any. Because it is X-linked, a mother can carry the gene change without symptoms; a genetic counselor can explain what this means for brothers and future children.^[1]

Forzinity is a US-approved medicine for Barth syndrome. On 17 October 2026 it was not found in the SFDA drugs list or the Abu Dhabi Department of Health drug code list, so in most Gulf countries it would need a named-patient import request by the treating hospital.^{[2, 3, 4, 5]}

US FDA-approved medicines for Barth syndrome

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.

Forzinity (elamipretide)
FDA-approved for improving muscle strength in adults and children with Barth syndrome who weigh at least 30 kg. It is a once-daily injection under the skin. Approved under accelerated approval, based on knee muscle strength.^{[2, 6]}
US FDA approval: 19 September 2025. 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.^{[3, 4]}
Country guides: Qatar, Egypt, India, Pakistan

How families in the Gulf can access these medicines

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.^{[5, 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 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.

Ask us about treatment for Barth syndrome

Tell us the medicine, the country, and how to reach you. We reply within one business day. On WhatsApp we usually reply the same day.

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Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

Frequently asked questions

Is there an FDA-approved treatment for Barth syndrome?

Yes. The medicine on this page has US FDA approval for a specific group of patients: Forzinity. The exact conditions are in the US label and are summarized above. Your specialist decides whether it fits.

Is Forzinity available in Saudi Arabia or the UAE?

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. A listing in Abu Dhabi does not show federal UAE status, which we could not verify.

How can a family in the Gulf get Forzinity?

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.

Does Reserve Meds diagnose or prescribe?

No. Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We do not diagnose, prescribe or decide what a regulator approves. Your specialist and hospital make the medical decisions.

Who can use Forzinity?

The US label covers adults and children with Barth syndrome who weigh at least 30 kg. It was approved under accelerated approval based on knee muscle strength, and continued approval may depend on a confirmatory trial.

Sources for the last answer: ^[6]

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.