2nd Annual

Patient Colloquium


October 30-31, 2024

Cambridge, Massachusetts


Days to the Event


About the Event

Please consider joining us for our 2nd annual n-Lorem Nano-rare Patient Colloquium.

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 are thrilled to be sharing more information about this day and a half event very soon.

2024 Nano-rare Patient Colloquium Agenda


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

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 conditionsPlease join us as we hear their diagnostic stories from the patients and their familiesWe 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.  This panel will feature four different patient/physician interviews that will be conducted by Brady Huggett and will be dispersed throughout the day and a half event. 


What it means go 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. This panel will feature executives from n-Lorem partners and supporters who believe in the mission and are committed to helping n-Lorem reach even more patients. 


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 todayOur physicians and institutional partners are key to ensure that not only are our patients cared for, but that they can access the treatments we offerWe 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 livesThis panel will include physicians from across the US to discuss their unique challenges and some of the innovative ways they have garnered institutional support for their patients. 


Innovative ways to support nano-rare

As n-Lorem scales to meet an ever-growing demand, new challenges ariseMany of these challenges will take innovative solutions and support from leaders and experts in a variety of different fieldsTogether as a community, we are creating a path forward for nano-rare patients and changing the way that genetic diseases are treatedThis panel brings together leaders and experts in areas where significant challenges exist to brainstorm how to overcome these challenges and advance treatments for even more nano-rare patients. 


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

This panel is back by popular demandn-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 share their diagnostic odysseys and what n-Lorem means for themWe will meet and learn about more nano-rare patient journeys to n-Lorem and the hope that n-Lorem brings to them and their families. 


Changing the world one patient at a time

At the onset, we wanted to engage renowned leaders in their fields to provide expert guidance as we navigate new territory, nano-rareWe believe that every patient deserves to have the best minds coming together to help them.  Our experts donate their time, their expertise and their insight for our patients and help us by serving on a number of committees that provide expert advice and guidance across all steps of the treatment pathway.  In this panel, our experts sit down to discuss why they believe in n-Lorem and together how we are truly changing the world one patient at a time.   

And more! — Additional presentations from physicians, experts, and n-Lorem staff to be announced


This Colloquium is hosted by Biogen

Biogen is a founding donor of n-Lorem and a leading global biotechnology company that has pioneered multiple breakthrough innovations. Biogen is advancing a pipeline of potential novel therapies across neurology, specialized immunology and rare diseases and remains acutely focused on its purpose of serving humanity through science while advancing a healthier, more sustainable and equitable world.


Are you traveling to this year’s event?

Download Travel Packet

Thank you!

To our generous supporters for this event


Do you or your organization want to sponsor the 2024 Nano-rare Patient Colloquium? Email [email protected]

Download Sponsor Packet

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.

We need your support

Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.

Follow us on social for updates on our latest efforts

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