Two-year-old has only 15 days of medicines left

The parents of twoyearold Aakriti Singh diagnosed with Spinal Muscular Atrophy ndash SMA type1 are worried about her next dose of medicine They were confident of getting central government financial aid from the Rs 924 crore assured in the budget for rare diseases as directed by the Delhi High Court But the parents are shocked to learn that the Supreme Court recently put a stay on the Delhi HC order putting SMA type1 patients and parents in limbo With no cure for SMA the medicines are imported and the prices are unaffordable which puts patients suffering from SMA at the risk of early deathWonder drugHer mother Kirti said ldquoRisdiplam a medication for definitive therapy manufactured by a multinational pharma company is one of the treatments for SMA This medication is lifesaving and must be given lifelong to reverse or stabilise the diseases manifestations and to prolong eventfree survival Aakriti requires one bottle every 30 days The approximate cost of each bottle including GST is Rs 620000 and the annual cost is nearly Rs 7500000rdquoAn earlier pic of baby Aakriti when she was diagnosed with SMASignificant improvementThe improvements observed through Risdiplam are significant Aakriti has achieved milestones such as sitting without support increased weightbearing in her legs and improved knee strength She can also scribble and recite poems like other children her age and she has shown no respiratory or swallowing problemsThe fearWhen asked what happens after the medicine is finished an emotional mother said ldquoAll her current milestones may regress which is not good for her Without medication she will be more prone to lung infections which could be lifethreatening As per the MNC pharma company scheme if we buy two bottles they give us three bottles without any cost So technically for Rs 12 lakh we get five bottles but this scheme is at the discretion of the MNC companyrdquoAccess to treatment toughldquoIn India approximately 4000 children are born with Spinal Muscular Atrophy SMA every year This genetic disorder leads to severe muscle weakness impacting essential functions like movement swallowing and even breathingrdquo said Alpana Sharma cofounder director of patient advocacy CureSMA Foundation India the largest parentled patient advocacy organisation floated by SMA childrenrsquos parentsSMA treatment in India currently faces significant hurdles Among the three globally available treatmentsmdashgene therapy Zolgensma Spinraza Nusinersen and Evrysdi Risdiplammdashonly Evrysdi is approved for use in the country ldquoIt is important to note that SMA patients are running against time The lifesaving medicines can not only arrest disease progression but also preserve functionalities in SMA patients We humbly request the government to give a new lease of life to our dying children by ensuring that all SMA patients get access to lifesaving medicine at the earliestrdquo Alpana saidldquoWe are still awaiting a response from the government and are hopeful of receiving National Rare Disease funds as per the law Given the exorbitant cost of the medication it is challenging for an average family to afford this treatment However does this mean denying the right to life to a little girl with so much potential and a future ahead Kirti concludedWhat is SMASpinal muscular atrophy SMA is a genetic condition that causes worsening muscle weakness There are five subtypes which range in severity and age of onset Therersquos no cure for SMA but certain therapies and medications can help manage symptoms