SMA is a group of hereditary diseases that progressively destroys motor neurons - nerve cells in the brain stem and spinal cord that control essential skeletal muscle activity such as speaking, walking, breathing and swallowing leading to muscle weakness and atrophy. Motor neurons control movement in the arms, legs chest, face, throat and tongue. When there are disruptions in the signals between neurons and muscles, the muscles gradually weaken, begin wasting away and develop twitching (called fasciculations).
There is no complete cure for SMA. Treatment consists of managing the symptoms and preventing complications.
Targeted Outcomes:
• Better outcomes for SMA patients and better Quality-of-Life for patients and caregivers
• Leadership position in SMA care
• Development of an SMA policy that can have a lighthouse effect on rare diseases policies across the nation
Objective:
• SMA, like other diseases in Group 3, requires life-long treatment. For now, parents and children have been left to fend for themselves, with crowdfunding being the only source of support for a bulk of them
• Given health is a state subject, there is an opportunity for states to provide comprehensive SMA care, covering care delivery, treatment and patient support. This could take the form of a State Level SMA policy, something that could benefit
• SMA patients
Action Plan:
• Kickoff meeting for both therapy areas with core group consisting of representatives from physicians, patient advocacy group and relevant policy makers
• MoU between O.T.G. and other stakeholders (Government - Dept. of Health and Family Welfare, HFI, FSMA etc.)
• Implementation of projects across both disease areas
• Conference - Rare Health Diseases - Bring in all the stake holders
• Technology Support
• Bring in Best Practices
