What Is Muscular Dystrophy?

Muscular dystrophy is a group of diseases within the same family that cause the deterioration of muscle mass and muscle cells, severe physical weakness, and commonly, the loss of mobility. Although there are different types of muscular dystrophy (categorized by the different muscle groups that they affect), they share a common cause: these types of illnesses are caused by “a mutation, or flaw, in a gene that interferes with the production of proteins needed to form and maintain healthy muscle. Such mutations may result in cells manufacturing a defective form of a particular protein, or insufficient (or completely absent) levels of protein.”

What Are the Different Kinds of Muscular Dystrophy?

Within this group of illnesses, some types reveal themselves during childhood and others are typically not diagnosed until adulthood. The different kinds of muscular dystrophy include:

• Becker muscular dystrophy – unique because it allows the voluntary muscles to function more effectively than other muscular dystrophies and usually has a later onset.
• Duchenne muscular dystrophy – a genetic disorder that causes progressive muscle degeneration and weakness from the mutated protein dystrophin, which (when healthy) keeps muscle cells intact.
• Emery-Dreifuss muscular dystrophy – Typically appearing by age 10, causing degeneration and weakness of the muscles of the shoulders, upper arms, and calf muscles. It can also cause the heart condition conduction block.
• Facioscapulohumeral muscular dystrophy – Genetic muscle disorder affecting the face, shoulder blades, and upper arms.
• Limb-girdle muscular dystrophies – A diverse disorder with multiple subtypes. Usually attacks the muscles around the hips and shoulders.
• Myotonic dystrophy – With a wide range of physical impacts, affects both muscles and organs.
• Oculopharyngeal muscular dystrophy – Rare and characterized by weakness of the eyelids and throat muscles. It can also cause muscle degeneration around the body midline and limb weakness.
• Titin muscular dystrophy – Rare form of muscular dystrophy caused by a mutation in the TTN gene, which leads to a slowing of the protein titin, leading to muscle weakness and degradation over time.

Types of Congenital Muscular Dystrophies

Congenital muscular dystrophies is a group of muscular dystrophy diagnoses that are typically diagnosed at or just after birth. These variations are defined by the wide variety of symptoms that they cause, including issues like joint stiffness or looseness, scoliosis, the degradation of respiratory function, intellectual disabilities, learning problems, eye issues or seizures. Some of the most common are:

• Bethlem CMD
• Fukuyama CMD
• Muscle-eye-brain diseases (MEBs)
• Rigid spine syndromes
• Ullrich CMD
• Walker-Warburg syndromes (WWS)

When children are born with severe forms of muscular dystrophy, they may never gain the ability to walk or attain other typical developmental milestones. When the disease occurs in older children and adults, they may lose their ability to walk. There is no cure for muscular dystrophy.

Other Complications from Muscular Dystrophy

Depending on the type of muscular dystrophy they are experiencing, children or adults could experience the degeneration of respiratory function, weakness with the muscles that help them swallow, intellectual disabilities, learning disabilities, eye defects, or seizures. Other issues may include the shortening of skeletal muscles or tendons around joints, scoliosis, and heart problems.

Is Muscular Dystrophy More Common in Males or Females?

Muscular dystrophy is caused by a deformity located on the X chromosome. Experts agree thar muscular dystrophy appears more frequently in males than females because they only have one X chromosome. Females possess two X chromosomes, and when the mutation is detected on one of the chromosomes, they do not present symptoms because their healthy dystrophin gene takes charge and their unhealthy X chromosome shuts down. However, they will continue to be carriers of the gene and could pass it on during reproduction. For women who may be a female carrier, doctors recommend genetic testing before conception.

When Should You Involve a Doctor?

If you notice a dramatic lack of muscle tone development in an infant, or if your infant or toddler experiences delays in reaching motor milestones like rolling over, sitting up, or walking, or fails to meet them at all, it’s time to have a discussion with your pediatrician.

The onset of variations of muscular dystrophy in adults commonly present with strong, rapidly developing signs of skeletal muscle weakness, including increased clumsiness or falling.

How Much Are Medical Expenses for Muscular Dystrophy?

The treatment cost for the wide range of muscular dystrophy types can vary dramatically. In the early stages, the average annual cost to treat a muscular dystrophy patient starts at around $20,000 before insurance. As the disease continues to progress, costs can reach more than $300,000, especially if reparatory function declines and a ventilator is required.

Financial Assistance for Muscular Dystrophy Patients with Help Hope Live

For families facing muscular dystrophy symptoms in a child or adult, Help Hope Live can assist with trusted fundraising to help families access medical assistance funds. Through community-based fundraising, our nonprofit organization is dedicated to helping those in need to raise funds for medical treatment costs associated with treatment for muscular dystrophy.

• Health insurance premiums, deductibles, and co-payments
• Medications
• Travel expenses for rehabilitation and treatment
• Relocation or moving expenses related to the diagnosis (such as in the case of moving to be closer to a rehabilitation center or hospital for treatment)
• Temporary housing due to relocation
• Mileage, tolls, and parking fees for visits to rehabilitation center or hospital
• Specialized wheelchairs and other durable medical equipment
• Specially equipped vans and durable medical equipment
• Home modification related to the diagnosis
• Home health care services
• Physical therapy and vocational rehabilitation
• Experimental treatments
• Assistive technology

How Is Help Hope Live Different from GoFundMe?

Help Hope Live differs from GoFundMe by:

• Verifying your medical need for complete donor confidence
• Enabling donations that are tax deductible
• Providing one-on-one fundraising help
• Managing all funds raised to protect your state-based benefits (funds raised are not considered personal income/assets to you)
• Paying bills directly, allowing you to focus on treatment and recovery

We are a nonprofit with more than four decades of fundraising experience and a 4-star Charity Navigator rating.

How Does Fundraising Work?

The fundraising process with our nonprofit starts with a few simple steps:

1. Complete a short campaign request at helphopelive.org/get-started/apply
2. We will contact you if community-based fundraising is a possible option
3. You’ll be paired with a Client Services Coordinator
4. Your Coordinator will provide you with one-on-one fundraising help, including personalized materials and guidance on how to rally your community, share your story on social media, reach

Does Community-Based Fundraising Work?

Here are a few Help Hope Live fundraising success stories:

“WE DID IT EVERYONE!! This is a one in a lifetime type of gift.”

Danny Stern has been using a wheelchair since he was ten years old as he experiences the progressive symptoms of muscular dystrophy. He earned his doctorate, passed the Bar, and secured employment as an associate attorney. When his accessible van became unreliable, Danny turned to Help Hope Live to fund a new van. He surpassed his $35,000 fundraising goal.

“Her motto for life is NEVER GIVE UP.”

Since 2009, the family of Scarlett Rose Landefeld has turned to Help Hope Live for secure, trusted, and experienced medical fundraising assistance to help with the costs associated with Scarlett’s spinal muscular atrophy (SMA) diagnosis. Scarlett has beat the odds since her second birthday, communicating with her eyes and engaging as fully as possible in the world around her. Fundraising ensures Scarlett’s community can rally to provide help, hope, and critical resources.

"It takes a village…Team Diane is here to support her."

Musician and teacher Diane Ellis survived a stroke during a jazz performance. “Team Diane” emerged to provide hope at a time of overwhelming need through a Help Hope Live medical fundraising campaign. With over $20,000 raised so far, fundraising helps Diane cover expenses including medical costs, pain management expenses, and ongoing therapy.