Clinical trials are essentially human biomedical or health-related research studies that follow a pre-defined protocol. These trials can be interventional (addresses whether experimental treatments or new ways of using known therapies are safe and effective under controlled environment) and observational studies (address health issues in large groups of people or populations in natural settings.)

By becoming part of a clinical trial, you not only proactively participate in your own health care and gain early access to cutting edge treatment, you also contribute to the treatment and research of the disease you are afflicted with.

I get questions all the time about clinical trials. Many parents of special needs children are overwhelmed when it comes to finding a clinical trial that is disease, age, and location appropriate. I watch the clinical trial scene religiously for an idiopathic/mitochondrial disease clinical trial that fits my daughter. The two websites that I find easiest to use, reliable, and always current are:

CenterWatch


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This site has a little of everything. It is an excellent source to find news, directories, analysis, and listings of clinical research. If you are unfamiliar with clinical trials, the site breaks down the process in an easy to understand manner. You can search clinical trials and find out the who, what, when, where, and how of each trial; search for drug information; review already completed trials; and sign up for e-mail alerts about specific clinical trials. Start your search at CenterWatch.

ClinicalTrials.gov 

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This site includes current information on federally and privately supported clinical trials for a wide range of diseases and conditions. Search by topic, keyword, disease, location, etc.. Studies are updated daily. At the moment, there are 91,384 trials listed on the site. ClinicalTrials.gov was developed by The National Institutes of Health (NIH) and The Food and Drug Administration (FDA…so users can rest assured that the listings are all legit. Start your search at ClinicalTrials.gov. 



If you have any questions, feel free to leave them in the comment section:)

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Science Translation Medicine has just published groundbreaking research on spinal muscle atrophy (SMA.)


SMA is a set of inherited muscle diseases that cause muscle to continuously degenerate and weaken. The disease requires extensive and ongoing medical care. The disease process usually ends with death. In fact, SMA is one of the most common genetic causes of death in childhood. Sadly, there is no known cure or effective treatment.

The research by University of Sheffield demonstrated a novel gene transfer system that has the promise of efficient therapeutic treatment for SMA patients. I will let you read the research process below, but the approach has the possibility of establishing gene therapy with the use of just a single injection. 

It is important to note that test was conducted using animals, but these findings undoubtedly put science that much closer to actual gene therapy treatments for SMA patients.

Research in depth:

New therapy offers hope to Spinal Muscular Atrophy (SMA) patients

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

New Novel Gene Treatment For Spinal Muscular Atrophy

To donate to SMA: 

Spinal Muscular Atrophy Foundation

 To support legislative action:

Legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, The SMA Treatment Acceleration Act