DMD Families Continue To Face Challenges In Accessing Promised Government Support

For most children, growing up means learning to run, play with friends, climb stairs and gradually become independent. But for children with Duchenne Muscular Dystrophy (DMD), these everyday milestones can slowly turn into struggles. A progressive genetic disorder, DMD gradually weakens muscles, robbing children of physical abilities they once took for granted.

Initially, children may stumble frequently, struggle to get up from the floor, climb stairs or keep pace with classmates. As the disease advances, many lose the ability to walk and become dependent on wheelchairs. Eventually, weakening of the heart and respiratory muscles can lead to life-threatening complications, requiring continuous medical monitoring and specialised care.

The disorder is caused by mutations in the dystrophin gene, which result in a deficiency of dystrophin, a protein essential for maintaining muscle fibre structure.

For parents, watching their children gradually lose their physical abilities is devastating enough. The challenge is compounded by difficulties in accessing sustained medical and financial support, despite several policy initiatives aimed at addressing the needs of patients with rare diseases.

Financial assistance has been announced, specialised centres designated and rare disease policies framed. Yet, families say access to medicines, specialised physiotherapy and comprehensive treatment remains a significant challenge.

Nineteen-year-old Arya Anand's journey illustrates the devastating progression of the disease. An active toddler who had even won first prize at a baby show in Gurugram, Arya was diagnosed with DMD at around five years of age in 2012. His parents had initially noticed that he frequently fell while running and appeared weaker than other children.

By the age of 10, he had become wheelchair-dependent. Today, his mother, Geetika Anand, says he is nearly 90% disabled and depends on his parents even for basic activities.

Akhilesh Tripathi, a 50-year-old shopkeeper from Jaipur and father of a son with DMD, says the emotional and financial strain is difficult to describe.

"'Traumatic' is an understatement for what we go through. Other parents talk about their children's future as they grow up. We cannot even afford to think that far ahead. In India, children with this condition rarely survive beyond 22 to 25 years because of inadequate access to medicines," he tells The Health Outlook.

The National Policy for Rare Diseases, 2021, provides for financial assistance of up to Rs 50 lakh per patient for treatment at designated Centres of Excellence, subject to clinical assessment and approval. However, parents say that while the provision offers important support, it does not fully address the continuing healthcare needs of children with DMD.

Tripathi points out that children with DMD require specialised physiotherapy throughout their lives to maintain muscle function and mobility for as long as possible. However, he says, the availability of physiotherapists trained in managing the condition remains limited at government-supported centres, leaving families with little choice but to arrange sessions at home.

"We have to call physiotherapists home and pay Rs 400-500 per session, often three times a week. This adds considerably to our monthly expenses," he says.

Although the government has increased the number of Centres of Excellence from eight to 15, parents say that strengthening the network needs to be accompanied by greater availability of specialised expertise, medicines and treatment facilities.

"Whenever we approach the centres, they say the medicines cost more than Rs 50 lakh and they neither have the expertise nor approval from the DCGI to administer them. They say they are helpless," Tripathi said.

Anand, too, says consultations often leave families with prescriptions for supplements, while access to disease-specific medicines and newer treatment options remains limited.

"Our concern is not that supplements have no role, but they cannot replace a comprehensive treatment plan involving appropriate medicines, physiotherapy, cardiac and respiratory monitoring and rehabilitation. These children need round-the-clock medical care," she said.

Meanwhile, a decline in rare disease funding has raised questions about the resources available to support treatment and care. Official data reveals that allocations have declined from Rs 82.87 crore in 2024-25 to Rs 32.73 crore in 2025-26, prompting concerns among patient advocates and medical professionals about the financial sustainability of Centres of Excellence across the country.

Although AIIMS Delhi and SGPGI Lucknow did not receive fresh funds, as they had unspent balances, patient advocates say sustained financial support will be important to ensure that centres can continue expanding access to care and expensive therapies for patients with genetic and metabolic disorders.

The financial burden further compounds the challenges faced by families. A March 2026 study published in the Journal of Postgraduate Medicine, titled Economic Burden of Duchenne Muscular Dystrophy from a Societal Perspective in Mumbai, India, examined 60 boys and their families at Mumbai's KEM Hospital.

It estimated the median annual economic burden at Rs 2.68 lakh per patient, equivalent to approximately Rs 22,360 a month. Significantly, 43.3% of the burden came from indirect costs, including lost earnings of carers. Three mothers had quit their jobs to care for their sons, while three fathers had changed workplaces to remain closer to home.

For parents, seeking medical support has also become an exhausting exercise in approaching authorities and navigating available support mechanisms. Several families have written to Prime Minister Narendra Modi and state government authorities, seeking access to newer therapies, clinical trials and domestic manufacturing of DMD medicines, but say they are still awaiting meaningful progress on their requests.

"We, the parents of children with DMD, have been seeking an appointment with Union Health Minister JP Nadda to apprise him of our difficulties, but we are yet to get one," Tripathi said. "We want the government to understand what families are going through and address the gaps in treatment and care."

The actual disease burden in India also remains uncertain. A review published in The Indian Journal of Paediatrics in 2026 by Dr Aradhana Rohil, Dr Gautam Kamila and Professor Sheffali Gulati of the Child Neurology Division, Department of Paediatrics, AIIMS, New Delhi, estimates that around four lakh to five lakh children in India could be affected by DMD. This is an estimate rather than a confirmed national patient count, with many cases potentially remaining undiagnosed or unreported.

In comparison, a written reply by the Union Health Ministry in the Lok Sabha on March 27, 2026, showed that only around 4,749 patients had been enrolled in the National Registry for Rare and Other Inherited Disorders across 23 centres since 2019. The registry had recorded 184 deaths among enrolled patients.

Although these figures are not directly comparable, the gap highlights the need to strengthen diagnosis, genetic testing, patient registration and disease surveillance.

Meanwhile, newer treatment approaches, including exon-skipping therapies and gene-based treatments, offer possibilities for selected patients. But access remains constrained by high costs, mutation-specific eligibility, regulatory approvals and safety considerations. Parents have sought access to medicines such as Exondys 51 and Vyondys 53, approved in certain countries for patients with specific genetic mutations, but say these remain largely inaccessible in India.

For children living with DMD, every year matters. While families await greater clarity on drug approvals, financial assistance and access to specialised treatment, the disease continues to progress. Strengthening the implementation of existing policies, expanding specialised care and improving access to emerging therapies could make a significant difference to the lives of these children and their families.