Patient Colloquium


October 12, 2023

Cambridge, Massachusetts

Register for Free!

Pre-registration ends October 5, 2023

Days to the Event


About the Event

Please consider joining us for our first n-Lorem Nano-rare Patient Colloquium. We believe that this meeting will be like none other, and we are thrilled to be sharing this special day with you. We will spend the day reviewing the progress we have made building the foundation and where we hope to be in the future. We will detail our processes and provide case studies that represent the challenges associated with discovering and developing an optimized personalized ASO medicine for a patient of one. Our patient families will share their diagnostic odysseys. Our partners will discuss how supporting nano-rare has helped their company culture. Our supporters will share the challenges ahead as the need only grows and more nano-rare patients find their way to n-Lorem. And so much more!

At this event, you will be joining a community of care that consists of patients and their families, physicians and institutional representatives, n-Lorem leadership team members, industry leaders, partners, donors and supporters. We have a full day planned with more than 20 speakers lined up for the event.


October 11, 2023
4:00 – 6:30 pm (EST)

Please join us the night before the Nano-rare Patient Colloquium for a delightful evening filled with mingling, small bites, and drinks. Meet community members and engage in meaningful conversations at the reception. We look forward to welcoming you with open arms the evening of October 11, 2023 at Biogen. See you there!

and Agenda

Thursday — October 12, 2023

7:30 – 8:15 am

Breakfast/Check In at Biogen

8:15 – 8:30 am

Welcome & Opening Remarks

8:30 – 9:15 am

Keynote Presentation:

The State of the Foundation;

Accomplishments and learnings over the past 4 years

n-Lorem exists because we believe that all patients deserve the opportunity to be treated, irrespective of the rarity of the disease or the economic status of the patient or family. In this presentation, Stan will provide an overview of our progress, what we are learning from all of our patients and what it takes to create a truly life-changing organization committed to offering free ASOs to patients for life.

9:15 – 9:30 am


9:30 – 9:45 am


9:45 – 10:15 am

Supporter Panel:

Helping n-Lorem do more

At n-Lorem, we focus on patients who have diseases caused by a single gene mutation that is found in no more than 30 patients in the world. We choose this patient population because today there is no path to commercial approval or commercial model that can address the needs of such rare patients and because we hope that we can raise sufficient donations to treat as many nano-rare patients as we are able to. Already the need has exceeded our expectations and we need more to do more. In this panel, some of our supporters discuss how as a community we can do more for nano-rare patients.



10:15 – 10:45 am

Foundation Panel:

Expanding the number of patients treated with high-quality ASOs

A core belief at n-Lorem is that every patient matters. The first dictum of therapeutics is to treat the patients who can be treated today while investing long term to identify better solutions for more patients. In this panel, we will discuss opportunities to expand the number of patients we can treat by working closely with appropriate institutions, patient advocacy groups and regulators outside of the U.S. We are committed to enhancing health care and changing the world one patient, one family at a time.



10:45 – 11:15 am

Patient Journeys:

The perilous journey to diagnosis and treatment for nano-rare patients

n-Lorem exists to serve the nano-rare patient community. The opportunity for ASO treatment provides hope to the hopeless and eventually, we hope, treatments for many. Hopelessness and despair that come at the end of a long and perilous journey to diagnosis are devastating. In this special panel, some of our nano-rare patients will share their diagnostic odysseys and what n-Lorem means for them.



11:15 – 11:25 am


Biogen’s Commitment to Nano-rare

11:25 – 12:00 pm

Partner Panel:

What it means to be a partner with n-Lorem for nano-rare

The challenges presented by nano-rare patients are far too complex for n-Lorem to solve even a fraction of the issues without support. Thus, our goal from inception has been to build as broad and effective a network of contributing stakeholders. In this panel, our partners in our community of care will discuss what it means to be a partner with n-Lorem. Including how partnering with n-Lorem provides their employees with a feeling of purpose.



12:00 – 12:10 pm

Hero of n-Lorem Awards

The ‘Hero of n-Lorem’ Award is a recognition given to an individual volunteer or organization that has made substantial contributions to n-Lorem and our nano-rare patients. This honor recognizes our ‘unsung heroes’, those who have gone the extra mile, taken time away from their schedules and families and leveraged their expertise and contacts to support n-Lorem’s mission to provide hope and potential help to nano-rare patients.

12:10 – 1:15 pm


1:20 – 1:40 pm


n-Lorem: Using Scientific Experience and Excellence to Drive Discovery and Development of Optimized ASOs

Every new patient represents a novel ASO discovery and development program. The essence of science is uncertainty and the process of discovering a novel medicine is complex and arduous, even for ASOs. At n-Lorem, we are industrializing the treatment of nano-rare patients because we believe that each patient deserves the very best ASO that we can discover and develop. In this presentation, we will provide data driven examples of hard choices that only expertise, a deep understanding of ASOs and decades of experience developing ASOs into a powerful drug discovery platform, can inform. Our patients deserve nothing short of the best that we can do.

1:40 – 2:00 pm


n-Lorem Clinical Infrastructure Assures Professional Management of our Patients

As our mission is entirely focused on helping nano-rare patients, the single most important area of progress will be to learn maximally from each patient. In this presentation, we will outline the infrastructure we have in place to ensure every step in the process be of the highest quality possible if we are to maximize the opportunity for benefit and minimize the risk of adverse events in our patients. We will discuss how we professionally manage exposure of each patient to an experimental medicine, accumulate, analyze and share data and the exciting data what we are seeing from our patients today.

2:00 – 2:15 pm


Integrated Operational Model: n-Lorem’s Scalable Solution for Nano-Rare Patients

2:15 – 2:30 pm



2:30 – 2:45 pm


2:45 – 3:10 pm

Physician Panel:

A physician’s perspective on n-Lorem and nano-rare

It is vitally important for n-Lorem to work with scientists and physicians to assure that patients are exposed to only optimized personalized ASO medicines. We are lucky to have world-class renowned physicians supporting our patients and helping us at every stage of the program’s development. In this assembly of medical professionals, our treating physicians discuss their experience with n-Lorem and how they support their patients throughout the process.



3:10 – 3:20 pm


Patient Empowerment Program at n-Lorem

One truly is “the loneliest number” and most nano-rare patients are exactly that: the single patient in the world with their unique mutation and disease. Whether a nano-rare patient is truly n-of-1 or a member of a tiny patient population, the patient is entirely isolated. In response, we are developing an array of pertinent support efforts that will seek to create a sense of community for nano-rare patients. In this presentation, we will discuss our Patient Empowerment Program and how we are creating a community of care.

3:20 – 3:45 pm

Supporter Panel:

Learning from nano-rare patients

Advances in science are incremental and each additional bit of knowledge contributes to the evolution of understanding. No area of scientific inquiry is more incremental than advances in the treatment of diseases. What can be learned by addressing the needs of patients with the rarest of mutations that cause a more common rare genetic disease is immediately applicable to the entire group of patients with the disease. In this panel, we will discuss how studying a patient or a few patients, even if they have the rarest of the mutations known to cause the disease, can expand our knowledge about the molecular causes of a disease and potential value of therapeutic interventions.



3:45 – 4:00 pm


Reforms needed to better care for nano-rare patients

In a developed economy, access to quality health care is a right, and at n-Lorem, we do our best for every single patient every single day. There are some obvious reforms needed that will be outlined in this presentation. We hope that more will join us, as we strive to enhance health care for nano-rare patients and change the world one patient, one family at a time.

4:00 – 4:20 pm

Closing Remarks