Invited to Washington, D.C.: Shaping the Future of Individualized Therapies

I was honored to be invited by Mila’s Miracle Foundation to participate in a high-level meeting with representatives from the U.S. Food and Drug Administration (FDA) in Washington, D.C.. Together with leaders from insurance companies, pharmaceutical firms, and lawmakers, we discussed one of the most urgent challenges in modern medicine: how to accelerate access to individualized therapies for children with ultra-rare diseases. A heartfelt thank you goes to Julia Vitarello, founder of Mila’s Miracle Foundation, for bringing together such a powerful and motivated group of stakeholders. Julia’s tireless advocacy has opened doors for so many families — and it was a privilege to be part of this important conversation. We made one thing very clear during the discussion: we must move forward. We cannot leave children with rare diseases behind. These are often very young children, suffering from devastating conditions with no existing treatment options. For them, time is not just precious — it is everything. All players in the system need to act: regulators, industry, payers, and legislators. We must reduce unnecessary delays, not add ever more layers of safety requirements, cost barriers, or bureaucratic complexity that render these life-saving programs practically or financially impossible. That does not mean compromising on safety — it means finding realistic and responsible pathways that match the urgency of these children’s situations. Valeria’s story, and the path we’ve taken with Valeriasen, is one example of what is possible. It’s now our shared responsibility to make sure this becomes the rule — not the rare exception. Yes, these therapies carry risks. Yes, side effects can occur. But when they are closely monitored, even these can be managed. Doing nothing is far more dangerous than moving these therapies forward with urgency and care. Every delay costs lives, potential, and time that these children simply don’t have. We owe it to Valeria — and to all the children like her — to solve this puzzle. To move boldly but responsibly. To act not out of fear, but out of hope, science, and love. That is Valeria’s legacy. And it must guide us forward.