Schedule of Event and Session Descriptions
We’re pleased to bring you the schedule of events for the 43rd Annual Family Conference. Please note that all sessions are held in Eastern Daylight Time. While planning your attendance, please note some sessions are specifically created for and limited to parents, grandparents and extended family, siblings, late onset, or all attendees. Please check back frequently as session times and content are subject to change. All times are EDT.
Thursday April 22, 2021
12:00-1:00 pm Welcome and Keynote Address
Please join us for our opening session of the 43rd Annual Family Conference. NTSAD is pleased to have Kyle Bryant and Sean Baumstark, hosts of the Two Disabled Dudes podcast. They will welcome us with their special brand of humor, while discussing their perspective on cultivating the courage to live beyond circumstance and laugh along the way.
This session is open to all attendees.
1:00–1:30 pm Break
1:30–3:00 pm Parent Peer Group
During this session parents currently caring for their affected children and those who are bereaved will gather in this support-based group setting to discuss the challenges in their lives as a family affected by rare disease.
This session is closed to outside participation and is only for parents currently caring for their affected child, or those who are bereaved.
Click here to join the Zoom (Closed Session)
1:30-3:00 pm Late Onset Support Group
This support session is designed for adults affected by Late Onset along with their spouses, partners, parents, or caregivers to come together as a group and discuss the challenges that come with living with Late Onset.
This session is closed to outside participation and is for Late Onset attendees only.
Click here to join the Zoom (Closed Session)
1:30–3:00 pm Grandparent and Extended Family Support Group
This support session is designed for grandparents, aunts, uncles, and others who are extended family members of affected children to discuss the challenges in their lives as affected by rare disease and how to support their loved ones.
This session is closed to outside participation and is for extended family members only.
Click here to join the Zoom (Closed Session)
1:30–2:30 pm Adult Sibling Support Group
This session is new this year. It is a chance for adult siblings, ages 18 and older to come together to discuss challenges as affected families, and how their experiences have shaped their lives since their sibling’s diagnosis.
This session is closed to outside participation and is for adult siblings only.
Click here to join the Zoom (Closed Session)
3:00–3:30 pm Break
3:30–5:00 pm Advocacy: Sharing Your Story
Join us in this new session focused on advocacy and empowering ways you can participate in advocacy efforts, big and small. You also will learn how to craft your story to make an impact.
This session is open to all attendees.
6:00–7:00 pm Virtual Family Lounge
The Family Lounge, an Annual Family Conference tradition, has gone virtual as well! Join us for a moment of downtime in the evening to get together, relax, connect, and chat as a group.
This session is open to all attendees.
Friday April 23, 2021
Each year at the Annual Family Conference, we devote an entire day to the latest advances in research, and we gather researchers, clinicians, members of industry and government to share their work, recent findings, and expertise. Be sure to visit the “Exhibit Hall” for viewing Posters and Abstracts. These sessions are open to all attendees.
12:00-1:30 pm Research Day Kickoff
Hear the latest NTSAD Research Strategy, how and why patient data is important, and features families’ voices and why they participate in research.
This session is open to all attendees.
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12:00 – 12:08 |
Introduction Oralea Marquardt, NTSAD Board Vice President |
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12:08 – 12:30 |
NTSAD’s Strategic Research Direction Fran Platt, PhD, Florian Eichler, MD, Gerry Cox, MD, PhD, Joan Keutzer, PhD |
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12:30 – 1:05 |
You and Your Data Staci Kallish to facilitate Panelists: Caitlin Nichols, PhD, AllStripes; Cynthia Tifft, MD, PhD, NHGRI |
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1:05 – 1:20 |
Family Voices Video This video will include voices of parents, caregivers and adults who have been involved in research in some way over the years. Hear what is has meant to them and the impact that they hope their participation has made. |
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1:20 – 1:30 |
Q&A – Previous speakers to participate |
2:00-4:00 pm Disease Specific Research Breakout Sessions
At the end of each disease-specific session (below), there is an opportunity for participants to ask questions. These sessions run concurrently and will be recorded. These sessions are open to all attendees.
Canavan Disease
Click here to Join the Canavan Disease Zoom
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2:00 – 2:05pm |
Introduction to Session Staci Kallish, DO, NTSAD Board President |
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2:05 – 2:25 |
Canavan Research: Antisense therapy David Pleasure, MD |
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2:25 – 2:45 |
Canavan Research update from the lab of Drs. Gao and Gessler Dominic Gessler, MD, PhD |
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2:45– 3:05 |
CANinform: Canavan natural history study Kathleen Kirby and Mike Kiefer, DPT |
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3:05 – 3:25 |
CANaspire: Aspa Therapeutics gene therapy clinical trial Jenny Laforet, MD, PhD and Kathleen Kirby, Aspa Therapeutics |
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3:25 – 3:45 |
Canavan Gene Therapy clinical trial, CureRareDisease LLC Paola Leone, PhD |
GM1 Gangliosidosis
Click here to join the GM1 Gangliosidosis Zoom
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2:00 – 2:05pm |
Introduction to Session Doug Martin, PhD, Auburn U |
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2:05 – 2:20 |
Gain Therapeutics research update Manolo Belotto, PhD, General Manager and President |
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2:20 –2:40 |
Idorsia Pharmaceuticals natural history study Jose Alcantara, Project Scientist |
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2:40 – 3:00 |
Azafaros drug development program Olivier Morand, CEO |
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3:00– 3:20 |
Lysogene gene therapy program Samantha Parker, Chief Patient Access Officer |
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3:20 – 3:40 |
Passage Bio gene therapy clinical trial David Weinstein, MD, MMSc, Passage Bio and Jeanine Jarnes, PharmD, U of MN |
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3:40 – 4:00 |
NIH / Sio gene therapy clinical trial Gavin Corcoran, Chief R&D Officer |
Infantile / Juvenile GM2 (Tay-Sachs and Sandhoff)
Click here to join the Infantile/Juvenile Tay-Sachs and Sandhoff Diseases Zoom
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2:00 – 2:05pm |
Introduction to Session Kevin Romer, Mathew Forbes Romer Foundation |
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2:05 – 2:25 |
Idorsia Pharmaceuticals natural history study Luba Trokan, MD, Director of Clinical Projects |
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2:25 – 2:45 |
Azafaros drug development program Ruben Giorgino, Head of Clinical Research and Development |
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2:45 – 3:05 |
Recursion Pharmaceuticals drug development program Joel Ellis, PhD, Director of Toxicology |
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3:05 – 3:25 |
Taysha gene therapies clinical trial Suyash Prasad, MBBS, MSc, Chief Medical Officer & Head of R&D |
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3:25 – 3:45 |
Sio Gene Therapies clinical trial Erika De Boever, VP Clinical Development |
Late Onset GM2 (Tay-Sachs and Sandhoff)
Click here to join the Late Onset Tay-Sachs and Sandhoff Diseases Zoom
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2:00 – 2:05pm |
Introduction to Session Gerry Cox, MD, PhD, NTSAD Board |
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2:05 – 2:15 |
LOTS Think Tank Steve Walkley, PhD, Albert Einstein College of Medicine |
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2:15 – 2:35 |
Gene therapy studies in sheep Heather Gray-Edwards, PhD, UMass Medical School |
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2:35 – 3:55 |
Medications Do’s and Don’ts Camilo Toro, MD, NIH |
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2:55– 3:05 |
Questions to ask your doctor about Clinical Trials Heather Lau, MD |
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3:05 – 3:25 |
IntraBio clinical trial Taylor Fields, Senior VP |
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3:25– 3:45 |
Sanofi Genzyme clinical trial Isabela A Batsu, MD, Global Project Head, Rare Disease and Rare Blood Disorder Development |
*Speakers as of 4/09/2021
6:00–7:00 pm Virtual Family Lounge
Join us again for a moment of downtime in the evening to get together, relax, connect and chat as a group, while we enjoy our traditional Pizza Party virtually!
This session is open to all attendees.
Saturday April 24, 2021
12:00–1:30 pm Health Management for Affected
In this detailed care-tip session, experienced parents and professions will share with families the myriad of factors involved in the care of affected children.
This session is designed for parents currently caring for their children, but is open to outside participation.
12:00–1:30 pm Living with Loss
In this session, bereaved families (including adult siblings, grandparents, and other extended family members) will come together in a support group style setting to discuss life after the loss of a child.
This session is closed and is for bereaved parents and extended family members only.
Click here to join the Zoom (Closed Session)
12:00–1:00 pm Late Onset Group
Kyle Bryant and Sean Baumstark, of the Two Disabled Dudes podcast, return to offer their perspective as they facilitate this discussion with Late Onset members to focus on the challenges of living with a life-altering diagnosis.
This session is closed to outside participation and is for our Late Onset attendees only.
Click here to join the Zoom (Closed Session)
12:00–1:00 pm Late Onset Caregiver Session
This session will offer a time for Late Onset caregivers to gather together and discuss the duality of their roles not only as caregivers, but as parents and spouses as well.
This session is closed to outside participation and is for our Late Onset caregivers only.
Click here to join the Zoom (Closed Session)
1:30–2:00 pm Break
2:00–3:00 pm Camp Snuggle Craft Time and Chat
Families caring for their child are invited to join in this interactive session with our Camp Snuggle leaders for a connecting time to chat and craft together.
This session is closed to outside participation and is only for families caring for a child.
Click here to join the Zoom (Closed Session)
3:00–3:30 pm Break
3:30–4:30 pm Caring for Your Canavan Child
Ilyce Randell, Max's and Alex's mom and founder of Canavan Research Illinois, will facilitate this session focused on the unique challenges of care management our Canavan families encounter from birth, through school age, and beyond.
This session is closed to outside participation and is for Canavan parents only.
Click here to join the Zoom (Closed Session)
3:30–4:30 pm Camp Sunshine Group Chat and Craft
Calling all siblings ages 4-11! Our Camp Sunshine leaders are hosting some together time with an interactive chat and crafting, so settle in and get ready for some special sibling fun.
This session is closed to outside participation and is for families of healthy siblings ages 4-11 only.
Click here to join the Zoom (Closed Session)
4:30– 5:00 pm Break
5:00–6:00 pm Family Planning and Carrier Screening
The question of expanding your family can be a particularly challenging one for carrier couples. In this session we’ll dive into the discussion of available options. We’ll also discuss ways to help educate other family members on the importance of carrier screening.
This session is closed to outside participation and is for parents only.
Click here to join the Zoom (Closed Session)
6:00–7:00 pm Virtual Family Lounge
Get those ingredients ready and join us again for a moment of downtime in the evening to get together, relax, connect and chat as a group, while we enjoy our signature NTSAD cocktails and mocktails!
This session is open to all attendees.
Sunday April 25, 2021
12:00–1:00 pm Commemoration
The commemoration ceremony is a special time where we honor our loved ones who have left us too soon. We invite all to join, listen to poems, and to light a candle for a their loved one when they see their tribute and photo(s).
This session is open to all attendees.
1:00–2:00 pm Break
2:00–3:00 pm Men’s Group
Specifically designed for dads, both caring for their children and those who are bereaved, this session is a chance for the dads of NTSAD to come together to discuss their roles as partners and fathers in their rare family.
This session is closed to outside participation and for dads of affected children only.
Click here to join the Zoom (Closed Session)
2:00–3:00 pm Women’s Group
Facilitated by Rare Mamas founder, Nikki McIntosh, this session is specifically designed for moms, both caring for their children and those who are bereaved, this session is a chance for the moms of NTSAD to come together to discuss their roles as partners and mothers in their rare family.
This session is closed to outside participation and for moms of affected children only.
Click here to join the Zoom (Closed Session)
2:00–3:00 pm Youth Group with Mentors
Specially designed for siblings between the ages of 12-18, the Youth Group session is a discussion among healthy siblings along with their older adult siblings on their challenges and roles within their rare family.
This session is closed to outside participation and is for youth group siblings (ages 12-18) only.
Click here to join the Zoom (Closed Session)
2:00–3:00 pm Late Onset Group
A combined session for our Late Onset members and caregivers this session will be a time to discuss the challenges associated with living with and caring for someone with a rare diagnosis.
This session is closed to outside participation and is for Late Onset members and their caregivers only
Click here to join the Zoom (Closed Session)
3:00–3:30 pm Break
3:30–4:00 pm Live Thank You Closing Session!
Please join us as we wrap up our conference with a thank you to all our attendees, sponsors, collaborators, and more. Together we’ll share final thoughts of the weekend as we connect to close out the 43rd Annual Family Conference.
This session is open to all attendees.
