Exploring Muscular Dystrophy and clinical trials

 

 

Types of Muscular Dystrophy

Muscular dystrophy is rare progressive neuromuscular disease which affects nerves, tissue and muscles causing them to waste away. Often muscular dystrophy is diagnosed at childhood, some types emerge in adulthood.   Due to the nature of the disease, patients experience muscle wastage and loss which can severely affect their movement ability and sometimes their ability to breathe.   Whilst there is no current cure for muscular dystrophy, there are a number of treatments available which can improve a patient’s quality of life. Clinical trial research continues to explore new treatments and procedures to help support patients and caregivers. There are over 30 different sub disease categories of muscular dystrophy. Some of the most common include:

Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a hereditary condition, caused by different mutations changes in DNA within the dystrophin gene which affects the muscles, causing them to become weaker and deteriorate over time. In Europe and North America, Duchenne Muscular Dystrophy affects 6 out of every 100,000 individuals.

Becker Muscular Dystrophy

Becker Muscular Dystrophy (BMD) is a genetic condition characterized by progressive muscle weakness. Whilst similar to Duchenne muscular dystrophy (DMD), it can have a later onset and milder symptoms compared to DMD. It is estimated that Becker muscular dystrophy occurs in approximately 1 in 30,000 male births.

Limb Girdle Muscular Dystrophy

Limb-girdle muscular dystrophy (LGMD) refers to a group of diseases that cause weakness and wasting of the muscles, specifically muscles of the shoulders, upper arms, pelvic area, and thighs. Estimates suggest that it affects around 0.8 to 6 people per 100,000 individuals worldwide.

Myotonic Dystrophy 

Myotonic dystrophy (DM) is a rare genetic multi-system disorder. This means its symptoms can affect, as well as muscle wastage, various organs and tissues such as the heart, lungs, the digestive system, eyes, hormone-producing glands and the brain. It is estimated to affect 1 in 2,100 people or over 3.6million individuals worldwide.

Young boy with Becker Muscular Dystrophy
Young man with Limb Girdle Muscular Dystrophy

Read our Muscular Dystrophy blogs

Disease Awareness
Genetic Testing
Myotonic Dystrophy
DM1

What is Myotonic Dystrophy

myTomorrows Team

18 Jan 2024

13 mins read

Duchenne Muscular Dystrophy
DMD
Adaptive Equipment
Accessibility

Adaptive Equipment for Duchenne Muscular Dystrophy

myTomorrows Team

18 Jan 2022

6 mins read

Knowledge
Duchenne Muscular Dystrophy
DMD
CRISPR
DNA

Duchenne Muscular Dystrophy and CRISPR Technology

myTomorrows Team

16 Nov 2022

8 mins read

Knowledge
Duchenne Muscular Dystrophy
DMD
Genetic Testing

Duchenne Muscular Dystrophy and Genetic Testing

myTomorrows Team

9 Dec 2022

9 mins read

Muscular Dystrophy Community support

Muscular Dystrophy Association (MDA)

MDA is a health nonprofit advancing research, care and advocacy for people living with muscular dystrophy, ALS, and related neuromuscular diseases.

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Parent Project Muscular Dystrophy (PPMD)

PPMD accelerates research, raises voices to impact policy, demand optimal care for every single family, and strives to ensure access to approved therapies.

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Akari Foundation

The Akari Foundation educate and empower the Hispanic community about rare diseases, help with resources, awareness, advocacy, and education, entirely in Spanish, specializing in Duchenne muscular dystrophy.

Visit webpage

What is a clinical trial and who can take part?

A clinical trial is a research study that examines on volunteers the effects of a medical treatment, device, or other intervention, which is not yet approved. Its purpose is to assess, prior to its potential approval, the safety and effectiveness of the treatment for a specific health condition before it becomes widely available. Besides focusing on treatment, clinical trials also explore new approaches to diagnosing and preventing diseases. 

Individuals with specific health conditions, such as muscular dystrophy, may qualify to participate in clinical trials. Researchers establish criteria for participation, which can be based on factors like age or the presence of other medical conditions. To determine eligibility, participants may need to answer questions or undergo a medical examination. Some trials also include healthy individuals without any known health conditions, while others may involve people with a family history of a particular disease or condition. 

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Why choose myTomorrows

We make it straightforward for patients and their physicians to discover and receive support with accessing clinical trials. We empower patients and their families to know and understand their options and make the best possible decisions regarding their treatment journey.

We've helped

11000+

patients access treatment

Cost to patients

Free

Active in

50+

countries worldwide

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Speak with our supportive team staff who are there to guide you through every step of your treatment option journey.  

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Frequently asked questions

1. How does myTomorrows help patients?

We empower patients and their physicians by offering a comprehensive overview on available options, whether it's participating in a clinical trial or considering an expanded access program.   Patients receive equal support – with a global reach, we provide referrals to trial sites and can support physicians with the expanded access application processes worldwide. To guide patients through this intricate process, our patient navigators act as a single point of contact throughout their entire journey. Learn more about how we help patients.

2. I am currently on a standard treatment. Can myTomorrows conduct a search at this time to explore potential non-standard treatment options?

It is important to remain on your current treatment, and to follow your doctor's advice. However, you don’t have to wait for your doctor to be referred to a clinical trial. Many people with a life-threatening disease actively look for clinical trials prior and during the treatment, to find more options to educate themselves about what is available.  

3. What is the role of Patient Navigators at myTomorrows?

Our Patient Navigators are an essential point of contact for patients to understand and find clinical trials and if available, expanded access programs.  They are a familiar contact for patients throughout their journey of choice; while they cannot give medical advice, Patient Navigators are trained to explain complex medical concepts to patients. We offer dependable, multilingual support to patients covering U.S. and European time zones.  Learn more about our services to patients here.

4. Can I quit a clinical trial?

Yes, you can quit a clinical trial at any time if you decide you no longer want to participate.

5. Do patients have to pay for clinical trials?

Patients usually do not have to pay for the majority of clinical trial costs. Drug manufacturers typically make the treatment in a clinical trial available free of charge. However, there may be other costs associated with participating in a clinical trial, such as additional medical appointments or travel expenses. Review any possible costs with the clinical trial staff and with your insurance provider before enrolling in a clinical trial. 

6. Who has access to my health data?

We treat all patient data highly confidentially and none is processed without your explicit consent. Our strict data policy restricts access to data to only qualified employees with direct operational responsibility for helping patients, and our platform and all our digital solutions are fully GDPR-compliant and ISO-27001 certified. More information is available in our privacy and cookie statements.  Do you have a question around data privacy, data processing, or data protection? Please reach out to dataprotection@mytomorrows.com 

7. Does myTomorrows provide medical advice to patients?

No, myTomorrows cannot provide any medical advice or medical services to patients. We kindly recommend that you discuss with your treating physician regarding any concerns or medical guidance in considering and selecting a clinical trial or expanded access program option. Treating physicians remain in charge of their patient’s treatment plan and providing any answers on specific medical questions. Our team can only support in providing information about clinical trials and expanded access programs that may be available to patients.

8. Is my patient’s data secure?

We treat all patient data highly confidentially and none is processed without your/their explicit consent. Our strict data policy restricts access to data to only qualified employees with direct operational responsibility for helping patients, and our platform and all our digital solutions are fully GDPR-compliant and ISO-27001 certified. More information is available in our privacy and cookie statements. Do you have a question around data privacy, data processing, or data protection? Please reach out to dataprotection@mytomorrows.com.

9. What is the cost of myTomorrows’ services for patients?

Our services of providing information about clinical trials and expanded access programs and supporting physicians with the expanded access application process worldwide are always free of charge for patients and healthcare professionals.
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