The Strive Foundation convened leading clinicians, caregivers and advocates in Islamabad to address Spinal Muscular Atrophy and sharpen Pakistan’s response to an urgent clinical and access challenge. The gathering highlighted practical emergency care, genetic counselling, treatment breakthroughs and a new digital patient registry designed to speed support to families across the country.
Strive Foundation reviewed a 12-year trajectory under Founder Muhammad Yasir Khan, tracing a modest start to a national platform focused on awareness, treatment access and caregiver support. Speakers noted how youth-led volunteers and targeted philanthropy have sustained outreach even as systemic barriers persist.
Dr Asim Ijaz, a UK-based emergency medicine consultant, urged emergency departments to adopt a simple stabilisation approach for SMA patients and introduced the SMART framework — Support breathing, Metabolic and hydration support, Airway management, Review baseline function and Trigger recognition — to reduce the risk of rapid respiratory deterioration. He emphasised that caregivers are an essential source of clinical information and warned against routine sedative use in fragile SMA patients, a point with direct implications for SMA Pakistan emergency protocols.
Dr Maryam Tariq outlined the genetic basis of the disorder, explaining that SMA results from deletion of the SMN1 gene and follows an autosomal recessive pattern. She described the four clinical types, from infantile onset to adult forms, and stressed the long-term psychological and physical toll on families, urging clinicians to combine technical care with compassionate support.
Dr Waseem Ur Rahman reviewed therapeutic advances that have changed prognosis for many children. He discussed spinal muscular atrophy treatments including Nusinersen, onasemnogene abeparvovec (Zolgensma) and risdiplam (Evrysdi), and credited Strive Foundation for facilitating access to these therapies in Pakistan. Success stories shared at the event showed children with Type 1 SMA achieving survival and developmental milestones previously considered unlikely, underscoring the impact of timely treatment.
Technology and transparency were also in focus when Waqar Hussain and Muhammad Uzair Khan demonstrated strivefoundation.co, Pakistan’s first automated SMA patient registration and sponsorship platform. The system allows caregivers to apply for funding and enables donors to follow medical updates and treatment progress, strengthening trust and targeting scarce resources where they are needed most.
Organisers reported that Strive has delivered 65 treatment cycles to 29 patients directly and contributed to nationwide efforts that have provided treatment to 102 patients overall. With 45 patients still on a waiting list, speakers called for sustained domestic funding and innovative solutions to international donation barriers, noting restrictions in cross-border banking and exclusion from crowdfunding platforms such as GoFundMe. Concerns were also raised about the withdrawal of some pharmaceutical operations from Pakistan, which has complicated access to essential medicines and research collaborations.
Leaders paid tribute to the foundation’s core team and early philanthropists who helped launch the programme. In closing remarks Muhammad Yasir Khan appealed for collective action to reach every registered child, invoking the Islamic injunction that saving one life is akin to saving all humanity as a moral imperative and urging renewed generosity as Ramadan approaches.
