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NEWS & UPDATES

TRIALS & STUDIES


Clinical Trials

If you are interested in taking part in a current or potential trial or study, it is important to let the doctors taking care of you know this – preferably your neuromuscular consultant or neurologist. They will let the centre/hospital conducting the trial know of your interest. It is sometimes possible the contact a centre and let them know yourself too, and they will then contact your local team.

This webpage will be updated whenever a new trial or study is approved and a centre or centres appointed.

Definition: a Clinical Trial is when a medical treatment, strategy or device is tested to see if it is safe and effective to use in affected patients.


AT132 for X-Linked Myotubular Myopathy – ASPIRO

Status: Active, not recruiting

Audentes Therapeutics, the San Francisco based biotechnology company have taken virus mediated gene therapy to clinical trial. Preliminary Results were encouraging and published in January 2018. Read the latest press release from Audentes Therapeutics here Read about this trial on clinicaltrials.gov Read more about Myotubular Trust’s grant awards to Dr Ana Buj Bello, who established proof of principle for this gene therapy. The first myotubular myopathy patient was dosed in this gene therapy clinical trial in September 2017.

Gene Therapy, the first treatment candidate for XLMTM, is explained here on the ASGCT website and in an educational video.


Natural History Studies

Definition: A Natural History Study follows a group of patients over time, to better understand how they are affected by a condition.


Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies (NatHis-CNM) MTM1, DNM2 & BIN1

Read about this study on clinicaltrials.gov     Status: Recruiting


Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) 2014 – 2017

Read about this study on clinicaltrials.gov     Status: Completed


A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM) – RECENSUS

Read about this study on clinicaltrials.gov     Status: Recruiting


A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects – INCEPTUS

Read about this study on clinicaltrials.gov     Status: Active, not recruiting


Other Links

Muscular Dystrophy UK Clinical trials FAQ page and information about Therapeutic development

CISCRP Patients In Clinical Trials Education page

MTM-CNM Family Connection Basic Overview of Drug Approval Process

Dr Suyash Prasad, Audentes Therapeutics Senior Vice President and Chief Medical Officer was interviewed about AT132 AAV Gene Therapy here.

American Society for Gene and Cell Therapy https://www.asgct.org/education/x-linked-myotubular-myopathy

 

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