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Spinal muscular atrophy (SMA): 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.

Spinal muscular atrophy: the short answer

The US FDA has approved four medicines on this page for spinal muscular atrophy, each for specific patients. Four of them are 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: Zolgensma, Itvisma, Spinraza and Evrysdi.
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; cell and gene therapies at a qualified center.
Price: Quote on request.

What is spinal muscular atrophy?

Spinal muscular atrophy (SMA) is a group of genetic diseases that damage and kill motor neurons, the nerve cells in the spinal cord and lower brain that control movement. As these cells die, muscles weaken and waste away, which can affect speaking, walking, swallowing and breathing.^[1]

Type 1 is the most common and most severe form. Babies usually show signs before 6 months of age, often cannot sit without help, and, without treatment, many die before age 2. Type 2 is usually noticed between 6 and 18 months; most children can sit but cannot stand or walk without help. Milder types begin later.^[1]

SMA is caused by changes in the SMN1 gene. In the US, a one-time intravenous gene therapy is approved for children under 2, an intrathecal gene therapy for patients 2 years and older, and two medicines that act on the SMN2 gene for children and adults: an injection into the spinal fluid and an oral solution.^{[2, 3, 4, 5]}

Spinal muscular atrophy in the Gulf

A study of 4,198 young Saudi volunteers found an SMA carrier frequency of 2.6%, and estimated a birth incidence of about 32 per 100,000 births. 27% of participants were children of first-cousin marriages.^[6]

In Qatar, 9 children with SMA aged 4 to 23 months received onasemnogene abeparvovec between November 2019 and July 2020. Motor scores improved significantly, and liver enzyme rises were seen without symptoms.^[7]

US FDA-approved medicines for spinal muscular atrophy

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.

Zolgensma (onasemnogene abeparvovec-xioi)
FDA-approved for children under 2 years with SMA caused by changes in both copies of the SMN1 gene. It is a one-time gene therapy given by intravenous infusion at a qualified center.^[2]
Given at a qualified treatment center. 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.^{[8, 9]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt, Turkey, India, Pakistan
Itvisma (onasemnogene abeparvovec-brve)
FDA-approved for adults and children 2 years and older with SMA and a confirmed SMN1 mutation. It is a one-time gene therapy injected into the spinal fluid at a qualified center.^[3]
Given at a qualified treatment center. Gulf registers: Saudi Arabia: not found by brand name 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.^{[8, 9]}
Spinraza (nusinersen)
FDA-approved for SMA in children and adults. It is injected into the spinal fluid (intrathecal) in hospital, with loading doses and then every 4 months.^[4]
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.^{[8, 9]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt, Jordan, Lebanon, Turkey, India, Pakistan
Evrysdi (risdiplam)
FDA-approved for SMA in children and adults. It is taken by mouth once a day.^[5]
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.^{[8, 9]}
Country guides: Saudi Arabia, UAE, Kuwait, Qatar, Bahrain, Oman, Egypt, India, Pakistan

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.^{[10, 11]} Once approval is in place, Reserve Meds arranges supply through a US-licensed pharmaceutical wholesale partner to the hospital pharmacy.

For Zolgensma and Itvisma. These treatments are made for each patient or need 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 that can give the treatment. A local hospital can only give it if the manufacturer agrees to supply that hospital and the regulator approves. 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 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 spinal muscular atrophy

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 spinal muscular atrophy?

Yes. The medicines on this page have US FDA approval for specific groups of patients: Zolgensma, Itvisma, Spinraza and Evrysdi. The exact conditions are in each US label and are summarized above. Your specialist decides whether any of them fits.

Is Zolgensma 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 Zolgensma?

It is given at a qualified center. Your specialist refers the patient, the center checks eligibility, and the family plans funding and travel. A local hospital can only give it if the manufacturer agrees to supply it and the regulator approves.

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.

Which SMA medicine is right for my child?

It depends on age, SMA type, genetics and earlier treatment. The US labels differ: Zolgensma is for children under 2, Itvisma for patients 2 and older, and Spinraza and Evrysdi for children and adults. Your neuromuscular specialist decides.

Why is SMA common in some Gulf families?

The Saudi carrier study found a carrier frequency of 2.6% and linked the higher incidence partly to consanguineous marriage. Carrier testing can help families plan.

Sources for the last two answers: ^{[2, 3, 4, 5]} ^[6]

Sources

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

Spinal muscular atrophy by country

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