Baby steps with Nemaline Myopathy

CE sheds light on how lives of people struggling with Nemaline Myopathy can be made easier through stronger support system and medical intervention
Baby steps with Nemaline Myopathy
Updated on
3 min read

CHENNAI: A young parent’s excitement knows no bounds to see their toddlers crawl, but what if the toddler’s movements are weaker, slower than expected? After a string of doctor visits and tests, the condition is given a name: Nemaline Myopathy.

The name might sound familiar to many. Recently, the former Chief justice of India, DY Chandrachud, talked about his daughters’ struggles, living with this disorder. For many families, this rare condition isn’t just a diagnosis — it marks the beginning of a new journey, with challenges and perspectives.

Understanding the condition

Nemaline Myopathy (NM) is a genetic muscle disorder that affects approximately one in 50,000 people, often showing symptoms from birth. Its name comes from the presence of “nemaline bodies” — thread-like structures that appear in muscle cells under a microscope. These threads might be harmless if not for the fact that they interfere with the muscle’s ability to function.

A neurosurgeon from the city says, “Nemaline myopathy is a genetic disorder that causes muscle weakness, often starting in the limbs. While there is no cure, treatments like physical therapy and respiratory support can help manage symptoms. Ongoing research into gene therapies may offer hope for better treatments in the future.”

Parents and caregivers often notice NM early in life:

  • Weak muscle tone and delayed motor skills: Babies might take longer to lift their heads, sit, or crawl.

  • Breathing difficulties: As the muscles responsible for breathing can also be affected, respiratory issues become a major concern, especially as children grow.

  • Feeding challenges and speech difficulties: In some cases, the muscles needed for chewing and speaking may be weak, adding another layer to the daily hurdles.

The journey to diagnosis can be emotional, as parents navigate specialists and tests. But once diagnosed, they find themselves in a world of ongoing care and cautious optimism.

There is no cure for Nemaline Myopathy, but supportive treatments are essential in helping patients achieve a high quality of life. Leading geneticists explain, “While we can’t reverse muscle weakness, we can give children the tools to maximise their potential.”

Key approaches

  • Physical therapy: A cornerstone of NM care, physical therapy helps patients retain muscle strength, mobility, and flexibility. According to physical therapists, each child has their own unique milestones.

  • Respiratory support: Some children require breathing aids at night or during illnesses. In severe cases, ventilators provide life-saving support.

  • Occupational therapy: Therapists teach techniques for everyday tasks like eating or dressing, helping patients gain independence over time.

Lifestyle adjustments and precautions

Living with NM requires day-to-day adjustments. Many families set up accessible environments at home, and prioritise nutrition and respiratory care. Avoiding physical strain is crucial, as overexertion can worsen muscle fatigue. Regular medical follow-ups become the norm, as doctors monitor each child’s progress and adjust therapies as needed.

Stories of resilience

For Nandhini* and Vivek*, the journey with their son Ishaan*, diagnosed with Nemaline Myopathy at nine months, has been one of patience and unexpected joy. “We had to learn to see the world at Ishaan’s pace,” Nandhini says with a smile. “When he finally managed to hold his toy elephant by himself, it felt like he’d conquered the world.”

Now three, Ishaan can’t run or jump like other kids, but he finds joy in his own way. Every morning, he insists on picking up a tiny toy car and placing it beside him at breakfast, grinning as if it’s a new achievement. “For us, those moments are everything,” Vivek shares. “Each small step he takes feels like a giant leap forward.”

Living with Nemaline Myopathy is not easy, but families facing it show a resilience that is nothing short of inspiring. Through therapy, medical support, and community, children with NM find their strength in different ways. And in a world built for hustle, they teach others the beauty of patience and the triumph of small steps.

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