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UK SMA Patient Registry

Launched by NEWCASTLE UNIVERSITY · Feb 28, 2020

Trial Information

Current as of June 26, 2025

Recruiting

Keywords

Spinal Muscular Atrophy Sma Neuromuscular Diseases Motor Neuron Disease Bulbo Spinal Atrophy, X Linked Kennedy Disease Spinal Muscular Atrophy With Respiratory Distress 1 Distal Spinal Muscular Atrophy Sma Type 1 Sma Type 2 Sma Type 3 Sma Type 4 5q Sma

ClinConnect Summary

The UK SMA Patient Registry is a research study aimed at better understanding spinal muscular atrophy (SMA), a genetic condition that affects movement and muscle strength. SMA can vary in severity and is caused by a change in a specific gene passed down from both parents. This study is open to all patients diagnosed with SMA, including those waiting for a diagnosis confirmed by genetic testing. There are no exclusions based on age or gender, so anyone with SMA can participate.

If you decide to join, you can easily sign up online through a secure website. After reading some information about the study and giving your consent, you’ll be added to the registry. Participants are encouraged to update their information every six months, helping researchers gather important data about SMA in the UK and Ireland. This ongoing research will help improve understanding and treatment of SMA for future patients.

Gender

ALL

Eligibility criteria

  • Inclusion Criteria:
  • All patients with a confirmed SMA diagnosis (or pending diagnosis) are eligible for inclusion. Diagnosis will be confirmed via genetic testing results
  • Exclusion Criteria:
  • There are no exclusion criteria for the registry

About Newcastle University

Newcastle University is a leading research institution located in the United Kingdom, renowned for its commitment to advancing healthcare through innovative clinical trials and scientific research. The university's Clinical Trials Unit plays a pivotal role in the design, coordination, and execution of high-quality clinical studies, focusing on a wide range of therapeutic areas. With a multidisciplinary team of experts, Newcastle University fosters collaboration between academia, industry, and healthcare providers, ensuring that research findings translate into meaningful advancements in patient care. Its dedication to excellence and ethical standards positions Newcastle University as a trusted sponsor in the clinical research landscape.

Locations

Newcastle Upon Tyne, , United Kingdom

Patients applied

0 patients applied

Trial Officials

Chiara Marini-Bettolo, MD, PhD

Principal Investigator

Newcastle University

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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