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A young boy from Colchester, Edward, who was diagnosed with spinal muscular atrophy (SMA) as a baby, has made remarkable strides in his development after receiving Zolgensma, the world’s most costly gene therapy drug. Now five years old, Edward can walk independently—a milestone that brought immense joy to his mother, Megan. SMA is a condition that impairs the production of a vital protein necessary for muscle growth, often leading to severe muscle weakness and life-threatening complications before the age of two if left untreated.
Edward’s journey began when he became one of the first children in England to be treated with Zolgensma through the NHS in 2021. This one-time gene therapy carries a list price of £1.79 million, though NHS England secured a confidential discount. Before receiving this treatment, Edward was on Spinraza, a drug requiring lifelong spinal injections. Megan described how her son transitioned from being lethargic and fragile as a baby to a lively, mischievous child full of personality. While Edward might still need a wheelchair, his mother emphasizes that his happiness is what matters most to their family.
Despite some challenges, such as a double hip replacement in October that temporarily slowed his progress, Edward continues to thrive. He has taken to swimming, managing to float independently—something particularly difficult for children with SMA. Over the summer, he even gained new experiences like jumping off a boat and riding a jet ski. Now attending school, Edward has gained many friends and participates in activities typical of a five-year-old boy. Doctors and medical professionals have been impressed by the improvements he has made, often visiting him during hospital stays to witness firsthand the benefits of gene therapy.
To support Edward’s ongoing rehabilitation, his family relocated to London to provide him with intensive physiotherapy, often up to five sessions weekly. Megan left her job in event management to care for Edward full-time and spearheaded fundraising efforts to supplement NHS care. Over five years, the family raised £170,000 to cover specialist physiotherapy and equipment, which Megan credits for much of Edward’s progress. Though the funds have nearly been exhausted, they continue to fundraise to maintain the level of care that has contributed so significantly to Edward’s quality of life. NHS England’s Prof James Palmer expressed optimism about the future, noting Edward is one of over 150 children benefiting from this innovative treatment and that advancements in gene therapies are expected to improve outcomes for many conditions in years to come
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