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Neurology medicines from the US: what is approved and how to access it

Alzheimer's, SMA, Duchenne, ALS, MS, myasthenia gravis and rare neurological diseases: checked against FDA labels on 17 October 2026.

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

The short answer

The US has approved new treatments across neurology, from antibodies for early Alzheimer's disease to gene therapies for spinal muscular atrophy and Duchenne muscular dystrophy and targeted therapy for SOD1 ALS. If a medicine is not registered in your country, the treating neurologist's hospital can request it for the named patient. Gene therapies are given at qualified centers.

  • Conditions covered: Early Alzheimer's, SMA, DMD, SOD1 ALS, MS, myasthenia gravis, NMOSD and rare disorders.
  • Medicines: Hospital named-patient or special access request.
  • Gene therapies: Given at qualified centers.
  • Price: Quote on request.

Early Alzheimer's disease

  • Leqembi (lecanemab): Alzheimer's disease, started at the mild cognitive impairment or mild dementia stage.[1]
  • Kisunla (donanemab): Alzheimer's disease, started at the mild cognitive impairment or mild dementia stage.[2]

Both labels limit treatment to the early stages of disease and describe imaging monitoring. The neurologist decides whether a patient qualifies.

Spinal muscular atrophy and Duchenne muscular dystrophy

  • Zolgensma (onasemnogene abeparvovec): one-time gene therapy for children under two years with spinal muscular atrophy and bi-allelic SMN1 mutations.[3]
  • Itvisma (onasemnogene abeparvovec-brve): gene therapy for spinal muscular atrophy in patients two years and older with a confirmed SMN1 mutation.[4]
  • Spinraza (nusinersen): spinal muscular atrophy in children and adults.[5]
  • Elevidys (delandistrogene moxeparvovec): gene therapy for ambulatory patients 4 years and older with Duchenne muscular dystrophy and a confirmed DMD gene mutation.[6]
  • Duvyzat (givinostat): Duchenne muscular dystrophy in patients 6 years and older.[7]
  • Agamree (vamorolone): Duchenne muscular dystrophy in patients 2 years and older.[8]

ALS, multiple sclerosis, myasthenia gravis and NMOSD

  • Qalsody (tofersen): ALS in adults with a SOD1 gene mutation (accelerated approval).[9]
  • Ocrevus (ocrelizumab): relapsing and primary progressive multiple sclerosis in adults, and relapsing-remitting MS in children 10 years and older weighing at least 25 kg.[10]
  • Briumvi (ublituximab): relapsing forms of multiple sclerosis in adults.[11]
  • Rystiggo (rozanolixizumab): generalized myasthenia gravis in adults who are AChR or MuSK antibody positive.[12]
  • Zilbrysq (zilucoplan): generalized myasthenia gravis in adults who are AChR antibody positive.[13]
  • Imaavy (nipocalimab): generalized myasthenia gravis in patients 12 years and older who are AChR or MuSK antibody positive, and warm autoimmune hemolytic anemia.[14]
  • Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase): generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy (CIDP) in adults.[15]
  • Enspryng (satralizumab): neuromyelitis optica spectrum disorder in adults who are AQP4 antibody positive.[16]

Rare neurological and metabolic disorders

  • Skyclarys (omaveloxolone): Friedreich's ataxia in patients 16 years and older.[17]
  • Daybue (trofinetide): Rett syndrome in patients 2 years and older.[18]
  • Miplyffa (arimoclomol): neurological manifestations of Niemann-Pick disease type C, with miglustat, in patients 2 years and older.[19]
  • Kebilidi (eladocagene exuparvovec): gene therapy for adults and children with AADC deficiency.[20]
  • Wainua (eplontersen): polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.[21]

How patients in the Gulf access these medicines

In Saudi Arabia, the SFDA Special Access Program lets a patient with a serious or immediately life-threatening condition receive a medicine that is not registered when no comparable alternative exists. The request comes from the manufacturer or sponsor, or from the licensed treating physician if there is no sponsor. We give you a written timeline estimate for your case before you commit.[22]

In Bahrain, NHRA Circular 41 of 2020 lets government and private hospitals import a non-registered medicine with the authority's permission before the purchase order, when the medicine is important and has no registered alternative in Bahrain, is registered with the Gulf Health Council, a GCC state or a reference regulator such as the US FDA, and is marketed in its country of origin.[23]

Infused medicines and those needing medical supervision are excluded from personal-use clearance in Saudi Arabia, so the hospital route applies. Gene therapies are on FDA's list of approved cellular and gene therapy products and are given at qualified centers.[24, 25]

How Reserve Meds helps

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help families and treating doctors understand the route, prepare the documents the regulator or hospital asks for, arrange supply from the US once approval is in place, and keep everyone updated. We do not diagnose, prescribe or decide what a regulator approves.

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. Your coordination fee is quoted in writing for your case before you commit. No hidden charges. We give you a written timeline estimate for your case before you commit.

Ask us about a medicine

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.

Include the country code, for example +966 or +971.
Message us on WhatsApp

No account needed. We reply within one business day. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

Frequently asked questions

Which FDA-approved medicines treat early Alzheimer's disease?

Leqembi and Kisunla, both for patients with mild cognitive impairment or mild dementia stage of Alzheimer's disease, as described in their US labels.

Is there a gene therapy for SMA?

Yes. Zolgensma is approved for children under two years with SMA and bi-allelic SMN1 mutations, and Itvisma for patients two years and older with a confirmed SMN1 mutation.

Is Qalsody fully approved?

Its US label states the SOD1 ALS indication is approved under accelerated approval, based on reduction in plasma neurofilament light chain, with continued approval possibly depending on confirmatory evidence.

How does a patient in Saudi Arabia get an unregistered neurology medicine?

Through the treating hospital's request to the SFDA Special Access Program, or referral to a qualified center for gene therapy.

Sources for these answers: [1, 2, 3, 4, 9, 22]

Sources

All sources were opened and checked on 17 October 2026. Rules and labels change, so the treating hospital or regulator confirms the current position before anything is filed.

  1. DailyMed: Leqembi (lecanemab) US prescribing information, version effective 21 July 2026 (accessed 17 October 2026)
  2. DailyMed: Kisunla (donanemab-azbt) US prescribing information, version effective 30 December 2025 (accessed 17 October 2026)
  3. US FDA: ZOLGENSMA product page (CBER) (accessed 17 October 2026)
  4. US FDA: ITVISMA product page (CBER) (accessed 17 October 2026)
  5. DailyMed: Spinraza (nusinersen) US prescribing information, version effective 31 March 2026 (accessed 17 October 2026)
  6. US FDA: ELEVIDYS product page (CBER) (accessed 17 October 2026)
  7. DailyMed: Duvyzat (givinostat) US prescribing information, version effective 21 November 2024 (accessed 17 October 2026)
  8. DailyMed: Agamree (vamorolone) US prescribing information, version effective 22 January 2026 (accessed 17 October 2026)
  9. DailyMed: Qalsody (tofersen) US prescribing information, version effective 13 November 2024 (accessed 17 October 2026)
  10. DailyMed: Ocrevus (ocrelizumab) US prescribing information, version effective 14 May 2026 (accessed 17 October 2026)
  11. DailyMed: Briumvi (ublituximab) US prescribing information, version effective 19 February 2026 (accessed 17 October 2026)
  12. DailyMed: Rystiggo (rozanolixizumab) US prescribing information, version effective 20 January 2026 (accessed 17 October 2026)
  13. DailyMed: Zilbrysq (zilucoplan) US prescribing information, version effective 3 February 2026 (accessed 17 October 2026)
  14. DailyMed: Imaavy (nipocalimab-aahu) US prescribing information, version effective 21 August 2026 (accessed 17 October 2026)
  15. DailyMed: Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase (human recombinant)) US prescribing information, version effective 15 July 2026 (accessed 17 October 2026)
  16. DailyMed: Enspryng (satralizumab) US prescribing information, version effective 22 June 2026 (accessed 17 October 2026)
  17. DailyMed: Skyclarys (omaveloxolone) US prescribing information, version effective 6 April 2026 (accessed 17 October 2026)
  18. DailyMed: Daybue (trofinetide) US prescribing information, version effective 4 September 2026 (accessed 17 October 2026)
  19. DailyMed: Miplyffa (arimoclomol citrate) US prescribing information, version effective 18 March 2026 (accessed 17 October 2026)
  20. US FDA: KEBILIDI product page (CBER) (accessed 17 October 2026)
  21. DailyMed: Wainua (eplontersen) US prescribing information, version effective 15 April 2026 (accessed 17 October 2026)
  22. SFDA: Special Access Program (SAP) guidance, version 1.0 (accessed 17 October 2026)
  23. NHRA Bahrain: Circular No. (41) of 2020, procedures for importing non-registered medicines (accessed 17 October 2026)
  24. SFDA: Conditions and Requirements for the Clearance or Exportation of Individuals for Personal Use, version 1.1 (accessed 17 October 2026)
  25. US FDA: Approved Cellular and Gene Therapy Products (accessed 17 October 2026)

Related

This page is general information, not medical or legal advice. It does not replace advice from your own doctor or the regulator.

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