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DNA strand

FDA approves trial to advance pioneering treatment for Hereditary Spastic Paraplegia (HSP)

The US Food and Drug Administation (FDA) has approved BlackfinBio Limited’s Phase 1/2 clinical trial for its novel adeno-associated virus (AAV) gene therapy targeting Hereditary Spastic Paraplegia (HSP) Type 47 (SPG47).
DNA strands

Sheffield spinout takes major step towards advancing pioneering treatments for Hereditary Spastic Paraplegia (HSP)

BlackfinBio Limited is a spinout company from the ±¬ÁϹ«Éç set to pioneer novel therapies for rare genetic neurological diseases.

Image of motor neurones

Groundbreaking EXPERTS-ALS study for MND treatments

The EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) project aims to rapidly screen potential drugs for evidence that they can slow down damage to the nervous system occurring in people with motor neuron disease
Professor Mimoun Azzouz holding a petri dish

Comment: Turning the biggest disappointment of a career into a new era for UK gene therapy manufacturing

Professor Mimoun Azzouz discusses career challenges before opening the Gene Therapy Innovation and Manufacturing Centre (GTIMC) at the ±¬ÁϹ«Éç.
Lumbar Puncture - treatment for MND
NIHR Sheffield Biomedical Research Centre

FDA approves drug which helps to slow progression of rare form of MND

The Food and Drug Administration (FDA) in the US has authorised the first approved drug for a rare form of motor neurone disease (MND) based on the results of a Phase 3 clinical trial completed at the ±¬ÁϹ«Éç.
Nerve cells

Scientists discover how to prevent death of nerve cells in most common genetic forms of MND and dementia

Scientists have found a novel way to block the transportation of mutant RNA and subsequent production of toxic repeat proteins which lead to the death of nerve cells in the most common subtypes of MND and FTD.