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#RareDisease
BBC 2d

A nine-year-old boy's life has been "fundamentally changed" thanks to approved treatments for spinal muscular atrophy (SMA), allowing him to reach significant milestones like starting mainstream school despite a grim prognosis. #SMA #RareDiseases #HealthcareInnovation #GB

Drug for rare condition 'fundamentally changed' Ramsgate boy's life
BBC 2d

A nine-year-old boy named Ezra, who has spinal muscular atrophy (SMA), has seen his life transformed by treatments recently approved for routine use in the NHS, allowing him to reach milestones that were once thought unattainable after a grim prognosis. #SpinalMuscularAtrophy #RareDiseases #HealthInnovation #GB

Drug for rare condition 'fundamentally changed' Ramsgate boy's life
BBC 2d

A nine-year-old boy named Ezra, who has spinal muscular atrophy, has seen a significant improvement in his life and the ability to start mainstream school following the NHS approval of nusinersen and risdiplam treatments, which were previously restricted but have now become widely available. #SpinalMuscularAtrophy #SMA #RareDisease #GB

Drug for rare condition 'fundamentally changed' Ramsgate boy's life