Donation for Adam

Thanks to you, there is now a chance for a recovery

With your help, the hope of a recovery is now within reach. Every donation brings Adam one step closer to life-saving treatment.

You can support Adam’s recovery on the following account:
“UNITED FOR ADAM” FOUNDATION (“ÖSSZEFOGUNK ÁDIÉRT” ALAPÍTVÁNY)
10300002-13877454-00014909
IBAN: HU46 1030 0002 1387 7454 0001 4909
BIC (SWIFT) MKKBHUHB

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Dear Supportive Community for Adam

We are deeply grateful for your generous donations and the unwavering support you have shown Adam during this challenging time. Your kindness brings hope and a chance for his recovery. Each contribution is a step closer to the treatment that can change his life. Thank you for being part of this journey. We truly appreciate your compassion and a helping hands.

With heartfelt thanks,
Adam’s Family

What do I need to know about Duchenne muscular dystrophy?

Duchenne muscular dystrophy is a genetic disease that is associated with increasing muscle weakness and loss of muscle mass. Muscle atrophy is caused by a genetic defect (gene mutation). It occurs mainly in boys, but is much less common in girls, who tend to carry the disease, i.e. they are not sick, but have the defective gene in their genetic make-up.

The disease is very difficult to detect before the age of one: the child may be slightly behind in motor development, but this is not usually alarming for parents. The symptoms become more noticeable later. Parents of boys aged 1-3 typically observe that their child:

  • Has difficulty getting up from a sitting or lying position
  • Falls frequently
  • Walks on tiptoes
  • Shuffles while walking
  • Has a waddling gait
  • Uses their knees to push themselves up when standing
  • Experiences muscle weakness affecting their posture, walking, and running
  • Learns to speak later than usual
  • Can only climb stairs with assistance
  • Has enlarged calf muscles
  • Suffers from muscle pain and stiffness
  • May have restricted joint movements due to muscle shortening
  • Experiences behavioral problems or learning difficulties
  • Later, heart muscle involvement may impact heart function
  • As muscles become weaker, breathing difficulties can develop.

The disease is caused by a faulty DMD gene. This gene encodes dystrophin, a protein essential for muscle regeneration and the formation of normal muscle structure. When a mutation occurs, either a defective dystrophin protein is produced, or no protein is made at all, leading to the degeneration of muscle fibers. As a result, muscle tissue is gradually replaced by fat and connective tissue.

The faulty gene can be inherited from a parent, but in about one-third of cases, the mutation develops spontaneously in the child. The disease follows an X-linked inheritance pattern, meaning that the mother passes on the faulty gene.

Complications of the disease can include:

  • Walking difficulties: By the age of 12-13, the child may become wheelchair-dependent.
  • Due to the wasting of shoulder and arm muscles, everyday activities become increasingly difficult over time, not just walking.
  • Joint contractures can develop as the muscles and tendons around the joints shorten, further increasing mobility limitations.
  • Breathing problems may arise due to the weakness of the respiratory muscles, and by the late teens or early 20s, the child may require mechanical ventilation.
  • Severe scoliosis (spinal curvature) can develop due to the weakening of the spinal muscles.
  • Heart complications (dilated cardiomyopathy) often emerge by the mid-teen years.
  • If the esophageal muscles are affected, swallowing difficulties can occur, leading to nutritional issues and an increased risk of aspiration pneumonia, making artificial feeding necessary.
  • Most patients do not survive beyond their 30s, often passing away due to heart or respiratory failure.