The Minister acknowledged the importance of Duchenne muscular dystrophy and the potential impact of givinostat. She highlighted that while some NHS trusts are offering early access, others have not due to concerns about cost-effectiveness and clinical evidence. The Minister stated that there is an urgent need for a more consistent approach across the country, and she has asked NHS England and other bodies to look at how they can support families in accessing this treatment where clinically appropriate and cost-effective. Acknowledges the profound impact of Duchenne muscular dystrophy and the urgency for new treatments. NICE assesses new medicines based on clinical effectiveness and cost-effectiveness, recommending two drugs for DMD: ataluren and vamorolone. Givinostat is being appraised by NICE with a meeting scheduled for July; if recommended, NHS England will fund it. Some patients receive givinostat through an Italfarmaco-led early access programme, but participation varies among trusts due to administrative and clinical costs. NHS England provides guidance on free-of-charge medicine schemes without directing trust participation. Facilitating information-sharing between participating and non-participating trusts is encouraged. For ambulant patients, eligibility criteria are set by the pharmaceutical company, with a trial underway for non-ambulant patients. Outside EAPs, the early access to medicines scheme (EAMS) helps people with life-threatening conditions gain early access to new medicines. The innovative medicines fund has provided £340 million for NHS funding of early access to recommended treatments, potentially speeding up givinostat access if approved by NICE. We acknowledge the significant challenges faced by patients seeking innovative treatments for rare diseases. In response to these issues, we have provided substantial support through various schemes designed to facilitate earlier access to potentially transformative medications. Specifically, we are reviewing the effectiveness of early access initiatives such as EAMS, ILAP, and IMF to ensure they adequately support individuals living with rare conditions like Duchenne muscular dystrophy. Annual meetings between NHS England, NICE, MHRA, patient advocacy groups, industry representatives, and clinical researchers will continue to address these issues, with the next meeting scheduled for summer this year. In addition, the 2025 action plan introduces new measures aimed at reducing the number of appointments required by families dealing with multi-system disorders and improving overall care coordination based on the CONCORD study definition. This work underscores our commitment to providing patients with access to innovative medicines while ensuring that such treatments offer value for money and are clinically safe and effective.