Patient at Royal Manchester Children’s Hospital is first in the North of England to receive life-saving gene-therapy drug

News posted 10 June, 2021

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A life-saving drug that can improve mobility in babies and young children suffering from a rare genetic condition has been given to a patient for the first time in the North of England at Royal Manchester Children’s Hospital.

Zolgensma is a one-off gene therapy that treats Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.

The latest data suggests that Zolgensma can provide improvement in motor function for young children with a specific type of SMA and prolong their lives.

Zolgensma, which has a list price of £1.79 million per dose, was made available on the NHS following a landmark deal struck with manufacturers Novartis Gene Therapies in March.

Royal Manchester Children’s Hospital (RMCH) is part of Manchester University NHS Foundation Trust, which is one of four centres approved for the use of Zolgensma for the treatment for Type-1 SMA. These are:

  • Evelina London Children’s Hospital (part of Guy’s & St Thomas’ NHS Foundation Trust) and with agreement to develop a sub hub at Great Ormond Street Hospital for Children NHS Foundation Trust
  • Manchester University NHS Foundation Trust
  • Sheffield Children’s NHS Foundation Trust
  • University Hospitals Bristol and Weston NHS Foundation Trust

The Trusts will be working as a national Multi-Disciplinary Team in collaboration with regional Neuromuscular Centres across the country to share expertise and experience for this new therapy.

Clinical teams at RMCH will assess patients individually for their eligibility and, alongside the family, consider whether treatment with Zolgensma is the best approach.

This recent treatment marks another significant milestone for gene therapies in the NHS and adds to the suite of Advanced Therapeutic Medicinal Products available at RMCH.

Dr Gary McCullagh, Consultant Paediatric Neurologist at RMCH said: “We are very pleased to be able to offer this treatment for our young patients affected by this debilitating, life-limiting disease. Although rare, Spinal Muscular Atrophy is still the leading genetic cause of death among young children. This new treatment will not only save lives, but will also improve quality of life for some children.”

Jane Valente, Medical Director at RMCH said: “The provision of this promising new gene therapy will come as very welcome news to families across the country. Here at Royal Manchester Children’s Hospital we look forward to providing this new service for our patients across the North West and beyond.”