Three disease-modifying therapies are fully funded by the NHS in England, following NICE's approval of all three. Here is what each one does and who can access it.
Spinraza was the first disease-modifying treatment for SMA to reach the NHS. It works by altering how the SMN2 gene's mRNA is processed — specifically helping to include exon 7 in the mRNA transcript — resulting in production of full-length, functional SMN protein. More SMN protein means motor neurons are better supported and disease progresses more slowly.
Spinraza is given by intrathecal injection, directly into the fluid surrounding the spinal cord, by a specialist at an SMA centre. A loading phase involves four injections over the first two months; maintenance injections are then given three to four times per year.
NICE recommends Spinraza for people with a confirmed diagnosis of SMA Types 1, 2 or 3, or pre-symptomatic SMA with 1–4 SMN2 copies. Not eligible if you've had successful treatment with Zolgensma, or are on permanent ventilation. Your specialist team assesses eligibility and monitors response.
The pivotal ENDEAR trial (Type 1 SMA infants) showed significantly improved motor function and event-free survival versus sham control. The CHERISH trial (Types 2 and 3) showed meaningful motor function improvements. Long-term SHINE extension data shows benefits maintained over multiple years.
Evrysdi works by the same principle as Spinraza — modifying SMN2 mRNA processing to produce more full-length SMN protein — but as a small molecule taken orally. It can be taken as a liquid at home once daily. For many families, the flexibility of home administration versus hospital injections is a significant quality-of-life advantage. Being a systemic therapy, it is distributed throughout the body and may address symptoms beyond the spinal cord.
NICE recommends Evrysdi for people aged 2 months or older with SMA Types 1, 2 or 3, or pre-symptomatic SMA with 1–4 SMN2 copies. Not eligible if Zolgensma has been successfully administered. Eligibility and treatment response is monitored by your SMA specialist team.
The FIREFISH trial (Type 1 infants) and SUNFISH trial (Types 2 and 3) showed clinically meaningful motor function improvements. The RAINBOWFISH study provided data on pre-symptomatic infants. Real-world evidence continues to emerge and is consistent with trial findings.
Zolgensma is a gene replacement therapy — the most fundamentally different of the three NHS treatments. Rather than modifying how SMN2 works, it delivers a functional copy of SMN1 directly into cells via an adeno-associated viral vector (AAV9). A single intravenous infusion is given at one of four national SMA treatment centres in England.
NICE recommends Zolgensma for babies with SMA Type 1 up to 12 months old, or pre-symptomatic babies up to 12 months with confirmed 5q SMA and up to 3 SMN2 copies. A National Multidisciplinary Team (NMDT) can also consider Zolgensma for older children with Type 1 within the EMA marketing authorisation (under 21 kg), reviewed case by case.
Some children treated with Zolgensma have developed liver enzyme elevations. Strict monitoring protocols are in place. Your clinical team will explain the monitoring schedule before treatment begins.
The STR1VE-US Phase 3 trial and SPR1NT trial (pre-symptomatic infants) showed striking outcomes — particularly in babies treated before or shortly after symptom onset. Long-term follow-up from the START trial showed motor function improvements maintained at 6+ years.
An intrathecal formulation of onasemnogene abeparvovec that delivers the SMN1 gene directly into the cerebrospinal fluid via lumbar puncture, rather than intravenously. This allows a much lower dose and is designed for older children, teenagers and adults who are too large for intravenous Zolgensma. Itvisma received FDA approval in the United States in November 2025, based on positive data from the STEER Phase 3 trial.
UK status: Itvisma has not been reviewed by NICE or authorised by the MHRA for use in the UK. There is no NHS commissioning of this treatment. Novartis would need to submit for MHRA marketing authorisation and a NICE appraisal before it could become available on the NHS.
Apitegromab is a myostatin inhibitor studied as an add-on therapy for people with Types 2 and 3 SMA already on SMN-targeting treatment. Rather than increasing SMN protein, it works by blocking myostatin — a protein that limits muscle growth and strength. Phase 2 data showed promise; a Phase 3 SAPPHIRE trial has been completed. No UK approval has been granted. Other pipeline areas include neuroprotective agents and combination therapy approaches.