The North America Rare Disease Summit brings together leading researchers, healthcare professionals, patient advocates, policymakers, industry experts, and rare disease patients and families. The summit emphasizes the power of collaboration and solidarity in facing rare diseases.
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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