A young boy, aged 12, who suffers from a rare muscle-wasting ailment has recently commenced a transformative treatment provided by the NHS, bringing relief to his family after a period of uncertainty. Ben Clarke has Duchenne Muscular Dystrophy (DMD), a severe genetic disorder that predominantly affects boys, leading to a progressive weakening and degeneration of muscles over time. He is one of many patients benefiting from givinostat, following its approval for use within select NHS Trusts through an early access initiative.
Despite the positive development, Ben’s father, Alex Clarke, emphasizes that the battle is ongoing, cautioning that other families may face tragic losses before being granted the same opportunity. Describing the moment Ben learned about the drug’s approval, Alex expressed to the Mirror how elated his son was, highlighting Ben’s happiness at the availability of the medication and his positive response to the treatment.
Givinostat is specifically designed to slow down the advancement of DMD and aid in preserving muscle functionality for an extended period. However, the drug’s approval is currently limited to boys who can walk, leaving non-ambulant boys without access.
Diagnosed with DMD at the age of four in 2017, Ben’s parents sought medical advice upon noticing troubling symptoms, ultimately turning to online research after a doctor’s suspicion of Duchenne. Alex, residing in Sonning Common, Oxfordshire, recalled the initial anguish upon learning about Ben’s diagnosis and the inaccurate prognosis they encountered online, causing immense distress and uncertainty about the future.
Now at the age of 12, Ben attends regular school and is described by his father as a beacon of light, brightening any room he enters. While Ben can still walk, he increasingly relies on a wheelchair for longer distances, with everyday tasks becoming more challenging for him.
The family spent over a year advocating for access to givinostat in collaboration with Duchenne UK through the ‘Time is Muscle’ campaign, after Ben’s trust initially did not participate in the early access program. The delays in the approval process have underscored the need for expedited reforms to ensure equitable access to vital treatments for rare diseases like DMD.
Alex stressed the importance of extending access to non-ambulant boys, highlighting the injustice of geographical disparities in accessing life-changing therapies. He emphasized the urgency in providing timely treatments to affected individuals to prevent irreversible deterioration and ensure equal opportunities for all impacted by such conditions.
