Mia Millan, a seven-year-old girl with epidermolysis bullosa, highlights the challenges faced by patients with this rare genetic disorder. Recent advances in medicine, including a gene therapy approved by the EMA that heals 70% of wounds within three months, give hope. Additionally, losartan, a longstanding hypertension medication, shows potential in treating skin lesions associated with the disease. While repurposing existing drugs can expedite treatment development, the necessity for valid clinical trials remains crucial for ensuring patient safety and efficacy before adopting these therapies in children.
Despite the promise of repurposed drugs like losartan, achieving clinical validation through rigorous studies is essential for ensuring efficacy and safety in pediatric patients.
The new gene therapy for epidermolysis bullosa shows a healing rate of 70% for wounds within three months, marking a significant breakthrough for affected patients.
Collection
[
|
...
]