What is Spinal Muscular Atrophy (SMA)?

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease that causes progressive muscle weakness. It is caused by a missing or faulty SMN1 gene, which leads to the loss of motor neurons in the spinal cord. SMA is the leading genetic cause of infant death.

What SMA treatments are available on the NHS in the UK?

Three disease-modifying treatments are currently funded by the NHS in the UK: Spinraza (nusinersen), Evrysdi (risdiplam), and Zolgensma (onasemnogene abeparvovec). Eligibility depends on SMA type, age, and clinical criteria set by NICE.

Is SMA screened for at birth in the UK?

Scotland includes SMA in its newborn bloodspot screening programme. England has received a formal proposal for inclusion in the NHS Newborn Blood Spot Screening Programme, which is currently under review.

What physiotherapy and standards of care are recommended for SMA?

International SMA standards of care recommend regular physiotherapy, respiratory monitoring, nutritional support, and orthopaedic surveillance. The specific interventions depend on SMA type and functional status (sitters, non-sitters, walkers).

What benefits can people with SMA claim in the UK?

People with SMA in the UK may be eligible for Disability Living Allowance (DLA) or Personal Independence Payment (PIP), Carer's Allowance, Universal Credit, Access to Work, and Disabled Facilities Grants (DFG).

🇬🇧 UK Community & Information

Living with SMA
in the UK

Independent information, real stories and practical support for people with spinal muscular atrophy, their families and carers across the United Kingdom.

What is SMA? NHS Treatments About Treat SMA

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SMA in the UK — Key Facts

~700People with SMA in England

1 in 40People carry the SMA gene

3NHS-funded treatments

2017First NHS treatment approved

✅ Fully approved on the NHS. Following NICE's final appraisal, Spinraza and Evrysdi are now permanently funded by the NHS in England for all eligible patients.

Where to start

Find what you need

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Just diagnosed?

Understanding your diagnosis is a lot to take in. Start here with the essentials — what SMA is, the different types, how it's diagnosed, and what typically happens next for your family.

Learn about SMA

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NHS Treatments

Three disease-modifying treatments — Spinraza, Evrysdi and Zolgensma — are currently funded by the NHS in England. Understand how each one works, who's eligible, and how treatment is delivered.

View treatments

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Living with SMA

From school and university to work, relationships and getting older — practical guides for every life stage, written with input from people actually living with SMA.

Life guides

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Benefits & Support

PIP, DLA, Access to Work and Carer's Allowance. Find out what financial support you and your family are entitled to, with rates updated for 2025/26.

Benefits guide

NHS England

Treatments currently funded

Three disease-modifying therapies are fully funded on the NHS for eligible patients, following NICE's final appraisal.

NHS — NICE Recommended

Spinraza

Nusinersen · Biogen

An antisense oligonucleotide that increases SMN protein production. Given by intrathecal injection at a specialist centre. NHS-funded since July 2019.

Route: Intrathecal injection (hospital) Frequency: Loading doses, then 3–4× per year Eligibility: Types 1–3, all ages

NHS — NICE Recommended

Evrysdi

Risdiplam · Roche

An oral liquid taken at home daily, increasing SMN protein by targeting the SMN2 gene. NHS-funded since January 2022.

Route: Oral liquid (taken at home) Frequency: Daily Eligibility: 2 months+, Types 1–3

NHS — NICE Recommended

Zolgensma

Onasemnogene abeparvovec · Novartis

A one-time intravenous gene therapy replacing the faulty SMN1 gene. NHS-funded for Type 1 SMA babies up to 12 months. Older children assessed by national MDT.

Route: Single IV infusion (specialist centre) Frequency: Once only Eligibility: Type 1, up to 12 months (NICE); older via MDT

Community

Voices from the SMA community

Too often, the conversation stops at what people with SMA can't do, which sadly is necessary when campaigning. However, to balance the narrative we want to celebrate what people with SMA are doing. #SMADidNotStopMe

This campaign isn't about pretending SMA is easy. It isn't about being inspirational. It isn't about ignoring the reality of living with a disability, having to navigate infrastructure and systems that are not designed with disabled people in mind.
It's about recognising achievement!