Days to the Event
About the Event
Please consider joining us for our second annual n-Lorem Nano-rare Patient Colloquium. Last year’s meeting was truly one-of-a-kind, and we are excited to expand this event and share two special days with you. Together, we’ll review the progress n-Lorem has made as we continue to grow our foundation, we’ll share new developments and explore where we aim to go in the future. We’ll dive into the knowledge we’ve gained at n-Lorem, the ways we’re enhancing our processes, and present case studies that highlight both the challenges and success stories in discovering and developing personalized ASO medicine for a patient of one. Our patient families will share their diagnostic odysseys and clinical experiences, while our partners will discuss how supporting nano-rare initiatives has enriched their company culture. Our supporters will address the growing challenges ahead as more nano-rare patients come to n-Lorem for help. Research physicians from various institutions across the country will provide insights into what we’re learning about neurological diseases—plus 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 more than 30 speakers lined up for the event.
2024 Nano-rare Patient Colloquium Agenda
Day 1 — October 30, 2024
Subject to change
1:00 – 1:45 pm
Check-in at Marriott
2:10 – 2:40 pm
Presentation
Opening remarks
President and Chief Executive Officer, Biogen
2:40 – 3:25 pm
Presentation
Providing hope for a better future, one nano-rare patient at a time
Stanley T. Crooke, M.D., Ph.D.
Founder and CEO, n-Lorem
3:25 – 3:50 pm
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
Brady Huggett
Features Editor at The Transmitter
Panelists
Ryan Strankowski
Patient, n-Lorem
Nelson Leung, M.D.
David L. and Colleen B. Kessenich Professor of Multiple Myeloma, Consultant in the divisions of Nephrology and Hypertension, and Hematology in the Department of Internal Medicine at the Mayo Clinic
3:50 – 4:15 pm
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
Brady Huggett
Features Editor at The Transmitter
Panelists
Kelley Dalby
Patient Mother, Director of Natural History and Diagnostics in Epilepsy at Praxis Precision Medicines
Olivia Kim-McManus, M.D.
Associate Clinical Professor, UC San Diego School of Medicine Dept of Neurosciences, Pediatric Neurologist and Epileptologist, Rady Children’s Hospital Precision Therapeutics Neuro-Interventional Program Director
4:20 – 4:40 pm
Presentation
Discovering patterns: learning from ‘n-of-few’ clinical trials in neurology
Jennifer Bain, M.D., Ph.D.
Associate Professor of Neurology and Pediatrics, Columbia University
4:40 – 5:05 pm
Patient Panel:
Hope realized; The culmination of effort, perseverance and belief in a better outcome
Susannah’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
Brady Huggett
Features Editor at The Transmitter
Panelists
Luke Rosen
n-Lorem Patient Father, Co-founder and Board Chair of KIF1A.ORG, Firefighter in the Sag Harbor Fire Department
Jennifer Bain, M.D., Ph.D.
Associate Professor of Neurology and Pediatrics, Columbia University
5:05 – 5:30 pm
Patient Panel:
Hope realized; The culmination of effort, perseverance and belief in a better outcome
Sloane’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
Brady Huggett
Features Editor at The Transmitter
Panelists
Megan Hedstrom
n-Lorem Patient Mom
Jennifer Bain, M.D., Ph.D.
Associate Professor of Neurology and Pediatrics, Columbia University
5:30 pm
Adjourn Day 1 of Meeting
Reception — October 30, 2024
5:45 – 7:30 pm
Reception
Please join us after Day 1 of the 2024 Nano-rare Patient Colloquium for a delightful evening filled with mingling, small bites, and drinks. Meet community members, engage in meaningful conversations, and bid on art pieces made by n-Lorem patients. We look forward to welcoming you with open arms at the Boston Marriott Cambridge. See you there!
Day 2 — October 31, 2024
7:15 – 8:00 am
Check-in and breakfast
8:00 – 9:45 am
Presentations
n-Lorem Team to present on the extraordinary progress being made for nano-rare patients
Sarah Glass, Ph.D.
Chief Operating Officer, n-Lorem
Laurence Mignon, Ph.D.
Executive Director, Clinical Development, n-Lorem
Konstantina Skourti-Stathaki, Ph.D.
Director, ASO Design and Discovery, n-Lorem
Julie Douville, Ph.D.
Executive Director, ASO Discovery and Development, n-Lorem
9:45 – 10:00 am
Q&A then short break
10:00 – 10:30 am
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
Elizabeth Berry-Kravis, M.D., Ph.D.
Professor of Pediatrics and Neurological Sciences at Rush University Medical Center in Chicago
Panelists
Margot A. Cousin, Ph.D.
Assistant Professor of Medical Genetics in the Dept. of Molecular Medicine, Director of the N-of-1 Therapeutics Program in the Center for Individualized Medicine, and a KL2 Scholar through the Center for Clinical and Translational Science at Mayo Clinic
Luke Hamilton, MS
Professor of Pediatrics and Research at the TCU Burnett School of Medicine, Research Data Science and Analytics team leader at Cook Children’s Medical Center
Horacio Kaufmann, M.D.
Axelrod Professor of Neurology, Professor of Medicine, and Professor of Pediatrics at New York University Grossman School of Medicine
Julie Ziobro, M.D., Ph.D.
Assistant Professor of Pediatrics in the Division of Pediatric Neurology at the University of Michigan
10:35 – 11:05 am
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
Natacha Gassenbach
Head of Corporate Affairs, Chief Communications Officer, Biogen
Panelists
Paul Compton
Chairman of Investment Banking, Barclays
Andrew Lo, Ph.D.
Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory
Alan Lotvin, M.D.
Chief Executive Officer, Sequel MedTech LLC.
John Maraganore, Ph.D.
CEO and Principal, JMM Innovations LLC.
David A Pearce, PhD
Chair, International Rare Disease Research Consortium
11:10 – 11:30 am
Presentation
New Business Models and Financing Strategies for Rare Diseases
Andrew Lo, Ph.D.
Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory
11:30 – 12:00 pm
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
Sarah Glass, Ph.D.
Chief Operating Officer, n-Lorem Foundation
Panelists
David Butler, Ph.D.
Chief Technology Officer, Hongene
Tammy Cooper
President, Nitto Avecia
Kurt Andersson
OligoProduct Specialist Genomic Medicine, Cytiva (Retired)
Stephen Wilson
General Manager, Charles River Senneville
12:00 – 12:15 pm
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
Kurt Andersson
OligoProduct Specialist Genomic Medicine, Cytiva (Retired)
Eugene Schneider, M.D.
Executive vice president and chief clinical development and operations officer, Ionis Pharmaceuticals
Neil Shneider, M.D., Ph.D.
Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University
12:15 – 1:00 pm
Lunch
1:00 – 1:20 pm
Presentation
What we are learning and how we can prepare for ASO treatments for rare neurogenetic conditions
Wendy K. Chung, M.D., Ph.D.
Chief, Department of Pediatrics, Boston Children’s Hospital
1:25 – 2:05 pm
Presentation
n-Lorem organizational updates
Stanley T. Crooke, M.D., Ph.D.
Founder and CEO, n-Lorem
Sarah Glass, Ph.D.
Chief Operating Officer, n-Lorem
2:10 – 2:30 pm
Presentation
Landscape of genetic diseases
Joe Gleeson, M.D.
Chief Medical Officer, n-Lorem Foundation
2:35 – 3:05 pm
Panel:
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-rare. We 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.
Moderator
Joe Gleeson, M.D.
Chief Medical Officer, n-Lorem
Panelists
Elizabeth Berry-Kravis, M.D., Ph.D.
Professor of Pediatrics and Neurological Sciences at Rush University Medical Center in Chicago, Co-Chair of STAR Committee (Study Treatment and Assessment Review), n-Lorem
Toby Ferguson, M.D., Ph.D.
Chief Medical Officer, Voyager Therapeutics, Member of ATTC (Access to Treatment Committee), n-Lorem
Eugene Schneider, M.D.
Executive vice president and chief clinical development and operations officer, Ionis Pharmaceuticals, Chair of DSMB (Data Safety Monitoring Board), n-Lorem
Neil Shneider, M.D., Ph.D.
Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University, Member of ATTC (Access to Treatment Committee), n-Lorem
3:05 – 3:15 pm
Break
3:15 – 3:35 pm
Presentation
What we are learning about ALS
Neil Shneider, M.D., Ph.D.
Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University
3:35 – 3:55 pm
Presentation
What we are learning about ion channels
Elizabeth Berry-Kravis, M.D., Ph.D
Professor of Pediatrics and Neurological Sciences at Rush University Medical Center in Chicago
4:00 – 4:10 pm
Presentation
Patient empowerment community report card
Amy C. Williford, Ph.D.
Sr. Director of Communications and Donor Relations, n-Lorem
4:10 – 4:40 pm
Panel
The perilous journey to diagnosis and treatment for nano-rare
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 share their diagnostic odysseys and what n-Lorem means for them.
Moderator
Amy C. Williford, Ph.D.
Sr. Director of Communications and Donor Relations,
n-Lorem
Robert “Bobby” Glenn
Patient Father and member of the Yellow Brick Road Project Board of Directors
Daphne Graskewicz-Prado
Patient Mother and Co-founder of Kinslow TUBB4A Foundation
Kayla Mansour
Patient Mother and supporter of The Rory Belle Foundation
Junko Shiozawa
Patient Mother, Journalist, President of Hawaii Web TV, and member of the CureDRPLA Board of Directors
Raena Vrtochnick
Patient Mother and Detective
5:15 – 5:30 pm
Final Q&A
5:30 pm
Adjourn Day 2 of Meeting
Host & Venue
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.
www.biogen.com
Boston Marriott Cambridge
50 Broadway
Cambridge, MA 02142
Travel
Are you traveling to this year’s event?
Thank you!
To our generous supporters for this event
Sponsor
Do you or your organization want to sponsor the 2024 Nano-rare Patient Colloquium? Email [email protected]
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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