2nd Annual

Nano-rare
Patient Colloquium

2024

October 30-31, 2024

Boston Marriott Cambridge

Cambridge, Massachusetts

We are delighted to share that 750 people joined us, both in-person and virtually. Our heartfelt thanks go out to the nano-rare community for making this event a success!

About the Event

During the 2024 Nano-rare Patient Colloquium, we reflected on n-Lorem’s progress as our foundation continued to grow, shared new developments, and presented our vision for the future. We explored the knowledge we had gained and outlined our processes that allowed us to efficiently develop optimal personalized ASO medicines. Our patient families shared their journeys through diagnosis and treatment, and our partners discussed how supporting nano-rare initiatives had enriched their company cultures. We also heard from our supporters about the growing challenges as more nano-rare patients sought help from n-Lorem. Additionally, research physicians from across the country provided insights into the latest findings on neurological diseases—and much more!

The event was an extraordinary display of solidarity and inspiration. We learned, laughed, connected, and even shed a few tears, emerging as a stronger and more united nano-rare community.

2024 Nano-rare Patient Colloquium

Day 1 — October 30, 2024

Opening Remarks

Presentation

Providing hope for a better future, one nano-rare patient at a time

Patient Panel:

Hope realized; The culmination of effort, perseverance and belief in a better outcome

Ryan’s journey and clinical experience

Our pioneering patients are paving a pathway for more nano-rare patients, providing valuable information on health and disease and experiencing, for the first time, targeted therapeutic intervention for their genetic conditions. Please join us as we hear their diagnostic stories from the patients and their families. We will follow these with case reports on these patient’s clinical experiences presented by the physicians who are so committed to caring for these patients.

Moderator

Panelists

Patient Panel:

Hope realized; The culmination of effort, perseverance and belief in a better outcome

Connor’s journey and clinical experience

Our pioneering patients are paving a pathway for more nano-rare patients, providing valuable information on health and disease and experiencing, for the first time, targeted therapeutic intervention for their genetic conditions. Please join us as we hear their diagnostic stories from the patients and their families. We will follow these with case reports on these patient’s clinical experiences presented by the physicians who are so committed to caring for these patients.

Moderator

Panelists

Presentation

Discovering patterns: Learning from ‘n-of-few’ clinical trials in neurology

Patient Panel:

Hope realized; The culmination of effort, perseverance and belief in a better outcome

Susannah’s and Sloane’s journeys and unique clinical experiences

Our pioneering patients are paving a pathway for more nano-rare patients, providing valuable information on health and disease and experiencing, for the first time, targeted therapeutic intervention for their genetic conditions. Please join us as we hear their diagnostic stories from the patients and their families. We will follow these with case reports on these patient’s clinical experiences presented by the physicians who are so committed to caring for these patients.

Moderator

Panelists

Day 2 — October 31, 2024

Presentation:

From dream to reality: The establishment of a scalable solution for the treatment of the nano-rare

Presentation:

ASO design, discovery and research innovation in ​ASO technology

Presentation:

Efficient nonclinical development processes leading to successful treatment of nano-rare patients

Presentation:

n-Lorem’s clinical experience paves the way for precision medicine using antisense oligonucleotides

Panel:

Building a nano-rare network and managing institutional challenges

Creating, developing and providing an individualized/personalized ASO medicine for an individual patient is only part of the challenge that faces n-Lorem and our patients today. Our physicians and institutional partners are key to ensure that not only are our patients cared for, but that they can access the treatments we offer. We are continually expanding our network to bring more physician and institutional partners together to work with us in getting our nano-rare patients the treatments that could change their lives.

Moderator

Panelists

Panel:

Innovative ways to support nano-rare

As n-Lorem scales to meet an ever-growing demand, new challenges arise.  Many of these challenges will take innovative solutions and support from leaders and experts in a variety of different fields.  Together as a community, we are creating a path forward for nano-rare patients and changing the way that genetic diseases are treated.

Moderator

Panelists

Presentation

New business models and financing strategies for rare diseases

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 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 and what more their organization can do to support nano-rare.

Moderator

Panelists

Heroes 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.

Awardees