Mumbai: Parents of children with rare disorder await relief from court

There are a little over 25,000 children with DMD in the state; all living with no viable cure in the country. Representation Pic

Parents of children with the rare disorder of Duchenne Muscular Dystrophy (DMD) who went to court in February 2023 have very little hope as their case has seen no progress. DMD is a severe, progressive genetic disorder that causes muscle degeneration and primarily affects boys. For the parents, each September 7, marked worldwide as the disease awareness day, serves as a stark reminder that a solution remains far off. There were parents of about 30 boys who were part of the petition before the Bombay High Court which sought directions to the Centre to provide free or subsidised treatment to children with such rare disease.

Anand Shah, one of the heads of the city's Parent Project Muscular Dystrophy, said they have received nothing substantial out of the petition as of now. "There are only more dates," he said. There are a little over 25,000 children with DMD in the state; all living with no viable cure in the country. The court has sought a response from the Centre and the next date is scheduled at the end of September. Shah's son was diagnosed with the disorder in 2009.

The symptoms of DMD appear in the ages of two and five and start with difficulty in walking, running, jumping, and frequent falls. Over time, this muscle weakness spreads. It first affects the legs and pelvis, and later the arms and respiratory muscles. By the time the child is in his teens, he is on a wheelchair.

Even the drugs that are available in countries like the United States can cost Rs 4 crore annually. "There is only supportive treatment available for DMD such as physiotherapy and occupational therapy in the country at the moment," said Dr Mukesh Agarwal, former head of KEM Hospital's paediatric department. Centre's 2021 National Policy for Treatment of Rare Diseases, which chose nine hospitals, including Mumbai's KEM Hospital, as Centre of Excellence for rare diseases has failed to yield much for these parents.

"The hospital (KEM) registers our names, takes medical history and tells us they would contact us in case there is a cure available. There is nothing else done. We have been requesting ICMR to fund studies for a cure. Many rejections have come our way. There is no hope for parents, especially from governments regardless of which party," Shah said. He added, "While worldwide there is recognition of families struggling with DMD every September 7, it is any other day here in the country."