“A rare disease fund has been set up by the government to help treat patients. In 2022-23, we provided assistance worth Rs 35 crore to 203 patients, a huge leap from zero funds three years ago. In 2023-24, this amount has increased to Rs 74 crore. In the current financial year, a budget of Rs 82.4 crore has been allocated, of which Rs 34.2 crore has already been disbursed. We realize that even this is not enough as we don't want to leave any patient behind,” said Dr L Swasticharan, Additional DDG with DGHS, Ministry of Health and Family Welfare (MOH&FW). Addressing the gathering at SMArtCon2024, the two-day national conference on Spinal Muscular Atrophy was held in Gurugram during the ongoing SMA Awareness Month.
Dr. L. Swasticharan announced that MoH&FW is actively considering setting up a special Technical Expert Group Focusing on spinal muscular atrophy, a rare and genetically inherited neurological disease that robs people of their physical strength by affecting motor nerve cells in the spinal cord. “Called Tech MSA, the group will advise Centers of Excellence for rare diseases across the country on what needs to be done about SMA and provide technical input. If we succeed in meeting the challenge of SMA, can solve, then the same model can be replicated for other rare diseases in the country.”
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“Awareness of rare diseases is also low among medical professionals, and most of them are not working in the field. There is a need for coordination between the government and the medical community to meet the challenge. We have rare diseases. A national policy and mechanism for including more 'orphan' diseases should come forward and help the government identify priority diseases for treatment as funds are limited There is a need to make medicines available and affordable for the patients. For this, the government is focusing on local research and production, supporting treatment and CSR funding. We are requesting special clinics for rare diseases Provide funds to establish where patients can go for treatment,” added Dr Swasticharan.
About 4,000 children are born with SMA every year in India. It is the number one genetic cause of infant death. The US FDA approved the first drug for SMA in 2016. Access, affordability, supportive care and delayed diagnosis remain major challenges. The ultimate solution lies in local research, but it will take decades. Meanwhile, we can't just leave existing patients to deteriorate and die.
“I urge the government to further increase budgetary support and give special attention to the treatment of rare diseases. SMA patients Who are saved for themselves at the moment. The treatment is so expensive that it is beyond the reach of almost every patient. We at Cure SMA Foundation strongly believe that successfully meeting the challenge of SMA will provide a model to effectively tackle other rare diseases in the country,” said Momita Ghosh, Co-Founder and Director, Family Support and Events, Cure SMA Foundation of India said.
“We have no other option.”
“We SMA patients succeed and give 200% to every effort we make, because we have no other option. We are very focused and determined. We sit in our wheelchairs for hours on end and work alone. Can do. We neither take breaks nor go out of the house. We can provide many times more value than an able bodied person Patients are successfully running businesses, setting up companies, topping CBSE board exams, and getting admissions in IITs.Giving us a chance to become mainstream and productive members of the society “We will not be disappointed,” said Rustam, a patient of SMA.
“The lack of treatment has made it extremely difficult for us to carry on with our lives,” says Roli Rastogi, 28, father of SMA and Akshit Rastogi, 26, who have SMA. The father of two says, “We feel deprived of going to public places, taking public transport services and accessing facilities because there is no provision for our children in these places. Lack of awareness about rare diseases. Because of which we feel isolated, the father of two says, “I request the government to give equal attention to patients like my children and social and Economically, our burden should be lightened.”
Parents of those struggling with SMA have called for an affordable and accessible treatment regimen. They have also emphasized on making public spaces accessible to everyone so that their children feel included in the society.
SMArtCon2024 was organized by Cure SMA Foundation of India to help build a sustainable ecosystem for SMA and other rare diseases. The participants included medical students, researchers, clinicians from places like Jammu, Coimbatore, Varanasi, Punjab, Odisha, Jaipur, and Delhi NCR, and numerous individual patients with SMA, along with their families from over 80 countries across India. More eminent doctors were involved. . It was jointly organized by the Academy of Child Neurology, Society for Indian Academy of Medical Genetics, Tata Institute of Genetics and Society, and Artemis Hospital (Gurugram).
Dignitaries at the event included Dr. DK Sibal, Deputy Drugs Controller, CDSCO; Dr. Debashesh Chaudhary, Director, Professor and Head of Neurology, GB Pant Institute of Postgraduate Medical Education and Research (GIPMER); Dr Shefali Gulati, Head of Pediatric Neurology, AIIMS Delhi; Dr. Ratna Dua Puri, Chairperson, Institute of Genomics, Sir Gangaram Hospital.