Our Daughters Are Running Out of Time We are the parents of Isla (9) and Siena (7) Treonze, two little girls from Bayonne, New Jersey, who are battling early-onset Facioscapulohumeral Muscular Dystrophy (FSHD) — a rare, progressive, and life-threatening muscle-wasting disease. FSHD is already recognized by the FDA as a fatal, life-threatening condition. Yet despite this, there is no pathway for our daughters — or any patient — to receive a promising treatment through expanded access. FSHD slowly and relentlessly destroys muscles. It takes away the ability to walk, lift arms, smile, swallow, and eventually, to breathe. There is no approved treatment. And without urgent action, our girls will lose everything this disease hasn’t yet stolen. The Treatment Exists — But No One Is Allowed to Have It A drug called AOC 1020, developed by Avidity Biosciences, is showing encouraging results in adults with FSHD. It was designed to target the root cause of the disease. But Avidity has chosen not to allow a single patient — child or adult — to receive it outside of their clinical trial. Even for children like ours who are rapidly deteriorating, there is no compassionate use, no expanded access, no right to try, no lifeline. Every Month is Another Loss We Can’t Get Back When adults wait for access, they lose time. When children wait for access, they lose their childhood that they’ll never regain — peer interactions, playground games, the strength to hug their parents, the breath to blow out birthday candles. For Isla and Siena, the wait means permanent and irreversible damage. By the time pediatric trials or policy changes arrive, it could be too late for them to benefit. What We Are Pleading We call on the FDA to: Use its authority to compel biotech and pharmaceutical companies to provide expanded access to children with fatal diseases when promising treatments exist.
Require public justification for any company refusing access in life-or-death pediatric cases.  We call on Novartis to: End Avidity’s across-the-board denial of expanded access.
Immediately provide AOC 1020 to severely affected children like Isla and Siena under FDA-regulated compassionate use. Why This Matters for Every Family Expanded access is a safe, regulated pathway. It has saved lives before without delaying drug approvals. When a company refuses expanded access to anyone, it’s not because the FDA forbids it — it’s because they choose to say no. And when the patient is a child with a fatal disease, that “no” is a death sentence. Help Us Save Our Daughters  We are asking you to stand with us. Tell Novartis, ow that Novartis has officially taken over Avidity Biosciences, I am asking the leadership responsible for medical access and compassionate use to please consider Isla and Siena’s case with urgency. I respectfully ask: Dr. Shreeram Aradhye – Chief Medical Officer Dr. Paul Aliu – Head of Global Governance Office (Compassionate Use Program) Dr. Liviu Niculescu – Chief Medical Officer, US Rob Rubinsky – Chief Market Access Officer, US Sonia Choi – Head of Communications & Patient Advocacy, US Dr. Marty Makary (Commissioner of the FDA), and the U.S. Food & Drug Administration: No child should be denied a fighting chance because of a company’s policy. Important Update: Immediate compassionate access program for Isla & Siena to Avidity’s investigational FSHD therapy (or therapies) under your AOC platform. 2. Transparent update on the timeline, eligibility criteria, expected launch date, and clinical development status of the FSHD asset(s) within Avidity’s pipeline. Why this matters: • Isla and Siena are living with FSHD now — each day, muscle strength and function decline with lifelong consequences. • Isla’s and Siena’s names — and the names of countless others with FSHD — remind us that lives hang in the balance. Our daughters are not statistics. They are little girls who still believe in tomorrow — a tomorrow that is slipping away. Every day without treatment means losing more strength, more independence, more childhood. This is not just about policy or protocol; it’s about the lives of children who deserve the chance to grow, to dream, and to live. We need action now. Because when you are a child with a fatal disease, tomorrow cannot wait. Please — sign and share this petition before it’s too late. Sincerely, Vera Treonze & Family Mother & Advocate for Isla (9) and Siena (7) Bayonne, New Jersey islasienafshd@yahoo.com