In addition to researching potential treatments for more common neurological conditions such as epilepsy or migraines, one key area of focus for Northwest Florida Clinical Research Group is a group of neurological diseases categorized as “rare.”
Let’s briefly explore what rare neurological disorders are, how they’re defined, and the critical role that clinical trial participants play in identifying emerging treatments.
We’ll also encourage you to contribute to our important work by joining one of our many ongoing clinical trials. We rely on your participation to develop the transformative therapies that enable affected patients to live fuller, longer, more productive lives.
What is a rare neurological disease?
A rare neurological disease is defined as a condition that affects less than 200,000 Americans.
Here are the key statistics on rare neurological disease:
- Researchers have identified in excess of 7,000 neurological diseases that qualify as rare.
- These affect over 25 million Americans and significantly contribute to mortality as well as negatively impact quality of life (QOL).
- Over 95% of rare neurological diseases have no currently known treatments.
Examples of rare neurological diseases include:
- Amyotrophic lateral sclerosis (ALS)
- Duchenne muscular dystrophy
- Huntington’s disease
- Denny-Brown syndrome
- Kleine-Levin syndrome
- Prion diseases
These conditions can impact one or more parts of the nervous system or associated systems and tissues such as the:
- brain
- spinal cord
- cranial nerves
- peripheral nerves
- neuromuscular junction
- nerve roots
- muscles
- autonomic nervous system
Rare neurological diseases don’t have to control your life. If you or your child is struggling with the symptoms of a rare neurological disease, new search studies could be an option for you.
Why do rare neurological diseases occur?
Rare neurological diseases may develop for one of several potential reasons. The underlying trigger might be:
- Inherited. There is often an identifiable genetic component to many rare neurological diseases. Accordingly, gene-based therapies hold therapeutic promise and are the focus of many ongoing clinical trials.
- Iatrogenic. Iatrogenic disease results from diagnostic procedures or treatments undertaken by a physician. This can include drugs that cause adverse, unanticipated side effects or surgeries.
- Postinfectious. Postinfectious illness following viral, bacterial, or fungal infection can occur in many systems of the body, including the nervous system.
- Unknown etiology. Sometimes, doctors can’t identify the origin of the disease. Improved diagnostic procedures may improve neurologists’ capacity to effectively identify and then treat neurological diseases with unknown causes.
What research groups fund and investigate new therapies for rare neurological disorders?
The unfortunate reality is that financial incentives often play a role in medical research – including which kinds of research get funded and which don’t.
Because rare neurological diseases by definition affect fewer numbers of people, they often receive less funding than clinical trials investigating treatments for more common ailments. That’s one of the reasons we at Northwest Florida Clinical Research Group (NWFLCRG) focus on rare neurological diseases.
However, several research institutions specifically fund and conduct research in these critical areas. Such examples of groups that take a leadership role in investigating new diagnostic procedures and emerging therapies for rare neurological disorders include:
- NIH National Institute of Neurological Disorders and Stroke
- Undiagnosed Disease Network (UDN)
- Rare Diseases Clinical Research Network (RDCRN)
Who qualifies for participation in a rare neurological disorder clinical trial?
Clinical trials are research studies designed to provide valuable data for a new or existing treatment, or treatment method, to determine if it is safe and effective for humans. Learn more about how clinical trials work.
Qualified participants include:
- Those diagnosed with a rare neurological disease.
- Those currently under the care of a physician for a rare neurological disease.
Additional criteria apply.
Those that qualify are seen by a board-certified physician at no cost, have access to possible new treatment for their condition and are compensated for time and travel. Learn more about the benefits of participation in clinical trials for patients.
In order to determine if a treatment is both safe and effective, testing must be conducted to provide statistical proof. According to laws and regulations, all clinical trials must be reviewed, approved and monitored by an Institutional Review Board (IRB) to ensure the ethical treatment of participants, and to ensure that all participants are fully informed about the study prior to agreeing to participate.
Contact Northwest Florida Clinical Research Group to become part of the solution
We are institutionally committed to identifying, exploring, and proving the effectiveness of emerging new therapies to treat rare neurological disorders that affect our Northwest Florida neighbors and the wider population.
To learn more about how we investigate new treatments for rare neurological disorders and how you can contribute to our efforts, contact Northwest Florida Clinical Research Group.
To keep up to date on the work we do, you can view current NWFLCRG studies. If you are interested in joining an ongoing study or if you have questions about clinical trial participation, apply online or call (850) 934-1299 for more info!
Clinical trials are research studies designed to provide valuable data for a new or existing treatment, or treatment method, to determine if it is safe and effective for humans. In order to determine if a treatment is both safe and effective, testing must be conducted to provide statistical proof. According to laws and regulations, all clinical trials must be reviewed, approved and monitored by an Institutional Review Board (IRB) to ensure the ethical treatment of participants, and to ensure that all participants are fully informed about the study prior to agreeing to participate.