Gene Therapy Patient Referral Pathways White Paper


Recommendations published to ensure fair and timely access to gene therapy clinical trials and treatments for Duchenne muscular dystrophy patients

The DMD Hub has collaborated with Cell and Gene Therapy Catapult, and the Northern Alliance Advanced Therapies Treatment Centre to identify and analyse the gene therapy patient referral pathways in the UK.

By highlighting best practice from across the UK, we propose a referral pathway for gene therapy clinical trials in the UK, to allow effective and equitable access to research for DMD patients.

As gene therapy is on the horizon for DMD as a licenced product, we considered how we can prepare for gene therapy licenced products in DMD as standards of care, using learnings and shared experiences from approved gene therapies in other neuromuscular disorders.

A set of recommendations for improved, standardised future practice, as well as referral pathways for gene therapy clinical trials and licenced products for DMD are proposed in the following report.

The report

You can read the full findings and recommendations in the report Evaluation of the Gene Therapy Patient Referral Pathways in the UK.


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