Treatment

It's not currently possible to cure spinal muscular atrophy (SMA), but there are treatments and support that can help. Research is ongoing to find new treatments.

Treatment and support aims to manage symptoms and help people with the condition have the best possible quality of life.

A team of different healthcare professionals will be involved in your or your child's care. They'll help make a care plan outlining the support and treatments you may need.

Medicines

Medicines that are used to treat some types of SMA include:

• nusinersen (Spinraza) – a medicine that targets the back-up copy of the faulty gene, given as an injection into the spine, every few months
• risdiplam (Evrysdi) – a medicine that targets the back-up copy of the faulty gene, given as a liquid once a day
• onasemnogene abeparvovec (Zolgensma) – a medicine that delivers a healthy copy of the gene that causes the condition, given as a single injection

Your treatment team will advise on whether these medicines are suitable for you or your child. This depends on the type of SMA and your or your child's age and symptoms. It's not always possible to have medicines to help treat SMA.

Read more about medicines for SMA from Spinal Muscular Atrophy (SMA) UK

Assistive equipment

If you or your child have difficulty moving or managing day-to-day activities, an occupational therapist or physiotherapist can provide advice and support.

For example, they can advise you about things such as:

• mobility equipment – including walking frames and wheelchairs
• supports for the arms or legs (splints or braces)
• shoe inserts that make walking easier (orthotics)

Find out more about household gadgets and equipment to make life easier.

Spinal Muscular Atrophy (SMA) UK has more advice for children, teenagers and adults about living with SMA.

Exercises and stretches

Exercises and stretches can help maintain strength and stop joints becoming stiff.

A physiotherapist can suggest some exercises to try.

The amount of exercise you or your child can do will depend on your condition, but it's best to try to stay as active as possible.

Breathing help

There are several treatments if you or your child have breathing problems because of SMA.

These include:

• chest physiotherapy and breathing exercises to strengthen the breathing muscles and make coughing easier
• a suction machine to help clear the throat if needed – this involves passing a thin, plastic tube to the back of the throat to suck away any mucus
• a machine to help with coughing and clearing mucus from the chest
• a machine that provides air through a mask (ventilator) put over your nose or mouth, or both

Your treatment team will advise on whether these treatments are suitable for you or your child. This depends on your or your child's specific breathing problems and needs. Your treatment team will give you training and support to use any equipment.

You or your child may be advised to have vaccinations against other conditions, including flu and pneumonia, to reduce your risk of becoming seriously ill from these infections.

Feeding and diet help

It's important for people with SMA, especially children, to get the right nutrients. This will help with healthy growth and development.

A dietitian can offer advice about feeding and diet.

If you or your child has difficulty feeding or swallowing, you may be referred to a specialist.

A feeding tube may sometimes be needed. Different types of tube can be used, such as:

• a tube passed through the nose and down the throat (nasogastric tube) – usually used for short-term feeding
• a tube attached directly to the stomach through the skin of the tummy (gastrostomy tube) – usually used for longer term feeding

Treatments for spine problems

Some children with SMA develop an unusually curved spine (scoliosis).

Treatments for this include:

• a specially made back brace to help support the back and encourage the spine to grow correctly
• spinal surgery – where the spine is straightened using metal hooks and rods, before being fused into place with pieces of bone

Research into new treatments

Research is being carried out into possible new treatments for SMA.

Find out more about new treatments on the Spinal Muscular Atrophy (SMA) UK website

You can ask your medical team about ongoing clinical trials into new treatments.

You can also search for SMA studies on the Be Part of Research website to find out about current research.