Nano-rare Colloquium Recap

My husband and I flew out to Boston for this year’s 3rd Annual Nano-rare Patient Colloquium after assembling a small “army” of support at home. My mother-in-law, our night nurse, and our nanny all pitched in so we could focus for a couple of days. It took a coordinated effort that rivaled a NASA launch, but somehow everyone got where they needed to be — and we made it onto the plane with both coffee and optimism intact – ready stand alongside the wider community of families affected by ultra-rare disorders. This event felt like more than science; it felt like connection, possibility, and showing up together.

Agenda Overview

• The conference spanned October 20–21, 2025, in Cambridge, Massachusetts. n-Lorem

• It was designed for a mix of audiences: patients and families, clinicians, researchers, biotech and industry partners, and patient-advocate organizations. n-Lorem

• Key themes running through the sessions included:

  1. Defining and understanding “nano-rare” conditions (often only a handful of people worldwide).

  2. Personalized therapeutic approaches: how science, data and patient insight come together.

  3. Diagnostics, trial design and regulatory pathways in ultra-rare settings.

  4. Manufacturing, delivery, and access challenges for treatments when patient numbers are extremely low.

  5. The role of patient families, advocacy, and community in shaping research and treatment development.

  6. Business, funding, and operational models tailored to the rarest of the rare.

• Beyond the formal talks, the agenda included opportunities for poster sessions, an evening reception, and community building — underlining that this wasn’t just about research, but about the people behind the research. n-Lorem

What It Means for Our Family & the Community

For us — and for the families reading Hope for SPTAN1 — this agenda signals both hope and realism. Hope: because the science is increasingly tailored, the community is visible, and the conversation is shifting. Realism: because the road from talk to treatment remains complex, and access + costs remain big questions.Overall, this event reminds us that even though our journey may feel solitary, we’re part of a broader movement. The kids we care for, the siblings we cherish — they all merit a voice in these spaces.

In his opening remarks, the n-Lorem Founder and CEO Dr. Stan Crooke set the tone by reminding us that “every patient matters.” He shared how n-Lorem’s mission — providing free, lifelong treatments for ultra-rare patients — has already reached real families. His focus was clear: progress comes when science and compassion move together.→ n-Lorem Foundation

Dr. Sarah Glass, Chief Operating Officer, explained that “nano-rare” conditions often affect only a few people on the planet, making each case truly one-of-a-kind. She discussed how researchers design personalized therapies, using each patient’s genetic data to create a match. Her message for families: connection between scientists and parents speeds discovery.

Another part of the agenda that stood out to me was the “Pathway to Treatment: from Diagnosis to Therapy” panel. The n-Lorem Clinical Team walked through how a diagnosis becomes a treatment plan — from gene sequencing to designing RNA-based medicine. They emphasized the importance of sharing data and building trust with families during every step. It’s nice when the scientists explain things in English instead of DNA!

As parents, we live in the in-between — between medical breakthroughs and bedtime stories, between science and survival. The Nano-rare Colloquium reminded us that progress is rarely neat, but it is happening. For every acronym and lab result, there’s a child who matters deeply — and a parent somewhere juggling logistics, love, and maybe just enough humor to keep it all afloat.