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Professor Volker Straub and his team at Newcastle University are developing important research tools that will support clinical trials for collagen VI-related conditions. This will strengthen the translational environment, helping to get potential treatments from the lab to patients in the clinic.
Professor Volker Straub and his team at Newcastle University are developing important research tools that will support clinical trials for collagen VI-related conditions. This will strengthen the translational environment, helping to get potential treatments from the lab to patients in the clinic.
Professor Volker Straub and his team at Newcastle University are developing important research tools that will support clinical trials for collagen VI-related conditions. This will strengthen the translational environment, helping to get potential treatments from the lab to patients in the clinic.

Our Q&A session with a neuromuscular clinical trial coordinator

Clinical trial coordinators play an important role in the successful running of clinical trials. So much so that drug companies are unlikely to select a hospital to run a trial if a clinical trial coordinator is not in post.
Clinical trial coordinators play an important role in the successful running of clinical trials. So much so that drug companies are unlikely to select a hospital to run a trial if a clinical trial coordinator is not in post.

About this article

Date Published:
9 November 2018
In this project, Professor Richard Piercy and his team will study dogs with naturally occurring Duchenne muscular dystrophy which may offer benefits over other currently used animal models of the condition. The researchers will carry out detailed investigations of skeletal and heart muscle function to better understand their symptoms and will assess their similarities to the human condition. The study should help the development of potential new treatments, ensuring that the best ones are taken to clinical trials as quickly as possible.
In this project, Professor Richard Piercy and his team will study dogs with naturally occurring Duchenne muscular dystrophy which may offer benefits over other currently used animal models of the condition. The researchers will carry out detailed investigations of skeletal and heart muscle function to better understand their symptoms and will assess their similarities to the human condition. The study should help the development of potential new treatments, ensuring that the best ones are taken to clinical trials as quickly as possible.
In this project, Professor Richard Piercy and his team will study dogs with naturally occurring Duchenne muscular dystrophy which may offer benefits over other currently used animal models of the condition. The researchers will carry out detailed investigations of skeletal and heart muscle function to better understand their symptoms and will assess their similarities to the human condition. The study should help the development of potential new treatments, ensuring that the best ones are taken to clinical trials as quickly as possible.
Clinical Research Fellow, Dr Hamilton and his team will investigate how the genetic changes that occur in myotonic dystrophy type 1 affect the severity of the brain-related symptoms associated with the condition such as impairment of thinking and excessive sleepiness. A better understanding of how genetic factors relate to the effects of myotonic dystrophy type 1 on the brain will help to identify new strategies for the treatment of these extremely debilitating symptoms. In addition, improved understanding of the variation in symptoms between individuals will be valuable in designing future clinical trials.
Clinical Research Fellow, Dr Hamilton and his team will investigate how the genetic changes that occur in myotonic dystrophy type 1 affect the severity of the brain-related symptoms associated with the condition such as impairment of thinking and excessive sleepiness. A better understanding of how genetic factors relate to the effects of myotonic dystrophy type 1 on the brain will help to identify new strategies for the treatment of these extremely debilitating symptoms. In addition, improved understanding of the variation in symptoms between individuals will be valuable in designing future clinical trials.
Clinical Research Fellow, Dr Hamilton and his team will investigate how the genetic changes that occur in myotonic dystrophy type 1 affect the severity of the brain-related symptoms associated with the condition such as impairment of thinking and excessive sleepiness. A better understanding of how genetic factors relate to the effects of myotonic dystrophy type 1 on the brain will help to identify new strategies for the treatment of these extremely debilitating symptoms. In addition, improved understanding of the variation in symptoms between individuals will be valuable in designing future clinical trials.

Muscular Dystrophy UK responds to Spinraza developers' Breakthrough prize

Submitted by Anonymous (not verified) on Thu, 10/18/2018 - 00:00

Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK:

“SMA is a life-limiting rare condition and patients desperately need access to Spinraza – the first and only treatment for this devastating condition. Without it, babies with the most severe form of SMA are likely to die, while others will irreversibly lose the ability to walk, crawl, breathe and swallow.

Dr Melissa Bowerman and her PhD student from Keele University will be investigating whether an already approved drug could be beneficial for spinal muscular atrophy (SMA).
Dr Melissa Bowerman and her PhD student from Keele University will be investigating whether an already approved drug could be beneficial for spinal muscular atrophy (SMA).
Dr Melissa Bowerman and her PhD student from Keele University will be investigating whether an already approved drug could be beneficial for spinal muscular atrophy (SMA).
Dr Gita Ramdharry and her PhD student at University College London (UCL) will investigate a self-management intervention for people with neuromuscular conditions. The focus is on living well with a condition, not just the medical needs.
Dr Gita Ramdharry and her PhD student at University College London (UCL) will investigate a self-management intervention for people with neuromuscular conditions. The focus is on living well with a condition, not just the medical needs.
Dr Gita Ramdharry and her PhD student at University College London (UCL) will investigate a self-management intervention for people with neuromuscular conditions. The focus is on living well with a condition, not just the medical needs.
Dr Gisèle Bonne and her team at the Myology Institute in Paris aim to understand what leads to the wide range of clinical severity in people with LMNA-Congenital muscular dystrophy (LMNA-CMD). This research could help to inform clinical care and identify new therapeutic targets.
Dr Gisèle Bonne and her team at the Myology Institute in Paris aim to understand what leads to the wide range of clinical severity in people with LMNA-Congenital muscular dystrophy (LMNA-CMD). This research could help to inform clinical care and identify new therapeutic targets.
Dr Gisèle Bonne and her team at the Myology Institute in Paris aim to understand what leads to the wide range of clinical severity in people with LMNA-Congenital muscular dystrophy (LMNA-CMD). This research could help to inform clinical care and identify new therapeutic targets.
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