On Tuesday, September 12, Delhi Chief Minister Arvind Kejriwal met with the family of 18-month-old Kanav, a boy diagnosed with a rare genetic disorder known as spinal muscular atrophy (SMA). The Delhi government, in coordination with the Central government, procured Zolgensma, one of the world's most expensive drugs, worth Rs 17.5 crore, used in the treatment of this disease.
Due to the Central government removing the import duty on the drug and the drug manufacturer agreeing to sell it at a reduced price, the family was fortunate to obtain the drug for Rs 10.5 crore. The child's parents raised the money through crowdfunding, facilitated by AAP MP Sanjiv Arora.
दिल्ली के डेढ़ साल के कनव को जन्म से ही SMA नाम की गंभीर बीमारी है। देश में अब तक ऐसे कुल 9 मामले ही हैं। इस बच्चे को 17.5 करोड़ का इंजेक्शन लगना था जो अमेरिका से आना था।— Arvind Kejriwal (@ArvindKejriwal) September 12, 2023
इस छोटे बच्चे को एक नई ज़िंदगी देने के लिए हमारे सांसद संजीव अरोड़ा जी और संजय सिंह जी के प्रयासों और जनता… pic.twitter.com/m8bkrffDt1
Kanav is the only child in the national capital diagnosed with this disease. In total, nine children in the country have been diagnosed with this genetic disorder.
Since this is a genetic disorder, there is currently no cure for SMA. The available drugs only ease the symptoms and prevent complications that might develop later in life.
In Kanav's case, the government procured the drug Zolgensma, which is gene therapy administered to children under 2 years old to prevent the progression of the disease.
In essence, Zolgensma is not a cure for SMA, and it cannot reverse the damage caused by SMA before treatment.
Zolgensma halts the progression of SMA and sustains the remaining muscle function needed for children to survive. The earlier Zolgensma treatment is administered, the better the preservation of muscle function.
Since it is an inherited disease, there is no way to prevent it. The most one can do is to undergo medical tests if the child exhibits symptoms.
Genetic testing, electromyography, and muscle biopsy are some tests used to detect SMA in children.
Treating SMA early in childhood prevents future complications and also enhances the quality of the child's life.