September 2022

In this Issue

Imagine & Believe 2022

Thank You Sponsors

Day of Hope 2022

Myra Sack Shares Lessons from Daughter Havi

Brady Manning Honors Brother Dylan with Beyond Birdies

College Scholarships for Healthy Siblings

In this Issue

Day of Hope

Imagine & Believe

GM1 FDA Meeting

Clinical Trials and Natural History Studies

Raise Rare Awareness in September

Gottlieb Memorial Fund College Scholarship

NTSAD’s 2023 Annual Family Conference

TORCH Awards Honor Blyth Lord

In this Issue

Letter from CEO Kathy Flynn

44th Annual Family Conference Highlights

Imagine & Believe

NTSAD Welcomes New Board President and Members and Recognizes Departing Members

Gray-Edwards Joins Advisory Council

NTSAD Honors Risa Asnen and CATS Foundation

Auburn University’s GM1 Research Has Positive Effect

Drive FORE Dylan

12th Annual Day of Hope

Thank You 2022 Conference Sponsors

Free Carrier Screening

Auction Items Needed!

In this Issue

Letter from Kathy Flynn

Passage Bio GM1 Clinical Trial

GM2 Clinical Trial Update

44th Annual Family Conference

International GM1 Day

Newborn Screening Legislation

Recognizing Courageous Mothers

NTSAD Community and Board Members Advocate for Patients and Families

ASGCT 25th Annual Meeting

Free at-home COVID-19 Tests

In this Issue

Letter from Kathy Flynn

44th Annual Family Conference

Imagine & Believe 2022 Honoree

GM1 Awareness Day

Granted: A Wish Story

Remembering Stew Altman

Auction, Auction!

44th Annual Family Conference

Myrtelle's Canavan Gene Therapy Receives Fast Track

STAT Act

TORCH Awards

Remembering Pioneers

Rare Disease Day

Importance of Carrier Screening

Continuing Nathan's Legacy

In this Issue

Letter from Kathy Flynn

65th Anniversary

Clinical Trial Updates

NTSAD's Research Funding

Altman Family Highlighted

Rare Disease Day 2022

Collaboration Enables Carrier Screening

MFRF Gala and Honoree

In this Issue

Thank You from Kathy Flynn

NTSAD Launches Next Round of Research Funding

Aspa's Gene Therapy for Canavan

Recognizing Seizures

Rare Disease Day 2022

NTSAD Parents Highlight the Power of Families to Advocate

NTSAD Mom Shares Her Love and Grief

Get Involved with NTSAD

*NTSAD’s COVID Guidelines

NTSAD announces the next round of research funding via the Research Initiative Program inviting proposals from academics and researchers.

Grants will be awarded to novel research projects that involve basic research, translational studies, or clinical studies that focus on Tay-Sachs, Canavan, GM1, and Sandhoff diseases and generate strong preliminary data that will attract significant funding from other sources.

In this Issue

Hello from NTSAD's New Leader

Aspa Canavan Clinical Trial

Sio's GM2 Gene Therapy

Fran Platt Elected Fellow of the Royal Society

Newborn Screening Advocacy

NTSAD Funds New Research

NTSAD Family Featured in USA Today

Granted: A Wish Story

Imagine & Believe

Support NTSAD Families

Gottlieb Scholarship Recipients

In this Issue

Imagine & Believe

NTSAD Hires Next Leader

GM1 Clinical Trial Updates

4th Annual LOTSS Think Tank

Grant for Tay-Sachs Sheep

Diagnostic Poster for Late Onset GM2

Day of Hope 2021

Rudness Family Interview

September 2021

In this Issue:

Imagine & Believe

Sanofi honors Dr. Kolodny

NTSAD's Research Director

Sue Kahn Recognized

Making the Rare Diagnosis

Day of Hope 2021

IntroBio's GM2 Clinical Trial

Lysogene Doses GM1 Patient

Sio's GM1 Clinical Trial

ASGCT's GM2 Guide

September Awareness

NTSAD Calendar

In this Issue

Aspa's Canavan Clinical Trial

Sio's GM1 and GM2 Gene Therapies

IntraBio Reports on Drug Development for GM2

GM1 Gene Therapy Guide

Imagine & Believe Sponsorship Opportunities

September: Tay-Sachs and Leukodystrophy Awareness Month

11th Annual Day of Hope

44th Annual Family Conference

Two Disabled Dudes Podcast

Jack Chesler’s Legacy

In this Issue

Aspa's Canavan Clinical Trial

Lysogene GM1 Gene Therapy Update

NIH, NTSAD, and Buryk Family Drive Late Onset Research

NTSAD Seeks Next Leader

Day of Hope

Imagine & Believe in October

Thanks and Welcome to Board Members

In this Issue

43rd Annual Family Conference

Sio's GM1 Gene Therapy Clinical Trial Six-Month Update

Mathew Forbes Romer Foundation Honors NTSAD

Profile of First Patient Dosed in Sio's Clinical Trial

Landsman Family Funds Canavan Clinical Trial

Thank You 2021 Conference Sponsors

In this Issue

Taysha GM2 Clinical Trial
Passage Bio GM1 Clinical Trial
Innovation Used in Tay-Sachs Gene Therapy
Late Onset FDA Listening Summary
2021 Annual Family Conference
Sheryl Sandberg Conversation
The Flysjö Family Story
We All Care for Rare
Motherhood and Parenting a Rare Child
Family Ancestry and Carrier Screening

In this Issue:

Sio Doses First GM2 Patient
Lysogene GM1 Clinical Trial
Sheryl Sandberg Conversation
2021 Annual Family Conference
Rare Disease Day is Feb. 28
GM1 Biomarker Results
NTSAD Seeks Research Director
Road to Tay-Sachs Gene Therapy

In this Issue:

Passage Bio GM1 Approval to Start Trial
Lysogene GM1 Approval to Start Trial
LOTSS Community & FDA
GM2 Natural History Study
Stem Cells and Sandhoff Mice
Gottlieb Memorial Scholarships
New Resources for Families
Annual Family Conference
The Mathew Forbes Romer Fdn.
Wear that You Care

In this Issue

Strength in Community
Sio Reports Positive Data
Passage Bio Approval
NTSAD Seeks Research Director
2021 Virtual Annual Family Conference
Diana's 25th Anniversary
Imagine & Believe T-Shirts

In this Issue:
Giving Thanks
Sio's GM2 Clinical Trials
Passage Bio's Potential
AllStripes' Data Partnership
Taysha and Invitae
Becca Heringer's Story
Imagine & Believe Again
Day of Hope Thanks
Giving Tuesday
AmazonSmile for NTSAD
The CARES Act

In this Issue
Imagine & Believe
GM1 Research Returns Hope
GM2 Clinical Trial Update
Canavan Families Webinar
Solace and Strength
Healthy Sibling Experience
Season of Hope

In this Issue:

• GM2 Late Onset Clinical Trial
• GM1 Clinical Trial Update
• AAV Gene Therapy Study
• GM2 Data Capture
• Rare Disease Summit
• Canavan Gene Therapy Webinar
• GM1 SSA Disability
• Partners during Pandemic
• Families Supporting Families
• Day of Hope with Team M&M
• NTSAD Team