Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

Lifeline Online: NTSAD Family News

August 2019

FAMILY SUPPORT

We know this time of year can be bittersweet for many families as the school year begins - a reminder of the milestones that are missed and those that will be missed. We want to take this time to say, "We see you and we're with you." As the dog days of summer begin to wind down, here are some photos of summers, past and present, to hopefully look back on with a smile. 

NTSAD's new vice president, Oralea Marquardt, wrote about this time of year for Courageous Parents Network when her son, William, would have been entering 4th grade. This year, William would have been in the 9th grade. You can read the full article here.

   
Segment borrowed from Family Caregiver Alliance
 
Below are guidelines adapted from the National Institute of Mental Health offering recommendations for dealing with depression. Set realistic goals in light of the depression. You may not be able to accomplish as much as you are accustomed to when you are feeling well.
  • Break large tasks into small ones, set some priorities, and do what you can as you can.
  • Try to be with other people and to confide in someone you know and trust; it is often better than suffering alone.
  • Participate in activities that may make you feel better, such as exercise, going to a movie or ballgame, or attending a religious, social, or community event.
  • Expect your mood to improve gradually, not immediately. Feeling better takes time.
  • It is advisable to postpone important decisions until the depression has lifted. Before deciding to make a significant transition—change jobs, get married or divorced—discuss it with others who know you well and can offer another view of your situation.
  • People rarely "snap out of" a depression. People who expect you to do this are misguided in their understanding of your condition.
  • Remember, positive thinking and the practice of new coping patterns will replace the negative thinking that is part of the depression. The negative thinking will be reduced as your depression responds to treatment.
  • Say “yes” to offers of help and engagement by family and friends who you know and trust.
  • Direct assistance in providing care for your loved one, such as respite care relief, as well as positive feedback from others, positive self-talk, and recreational activities are linked to lower levels of depression. 

Read the full article from the FCA newsletter here.


A Resource from National Organization for Rare Disorders (NORD): Rare Caregiver Respite Program

The Respite Program provides financial assistance to enable the caregiver a break to attend a conference, event or simply have an afternoon or evening away from caregiving. Financial assistance will be granted up to $500 annually for those who qualify. FOR MORE INFORMATION ABOUT THIS PROGRAM, CONTACT NORD AT: 203.616.4328 or email This email address is being protected from spambots. You need JavaScript enabled to view it.


Benton's Family Assistance Fund and Emma's Fund for Families are two funds within NTSAD that are there to provide families with help with equipment, caregiver help, a weekend away, a photography session, and more. Be sure to check it out as they are both additional strategies to help your family.

 

RESEARCH

BIG NEWS: GM1 Gangliosidosis Type II gene therapy trial now recruiting at NIH. Read more about this exciting news from the NIH directly here.


IntraBio Webinar

NTSAD hosted its first webinar with a presentation by IntraBio's Senior Vice President, Taylor Fields, and Dr. Tatiana Bremova from University of Bern in Switzerland. They spoke about their work and the current clinical trial for GM2 Gangliosidosis that is recruiting right now adults over the age of 18.
 
A recording of the webinar can be seen here along with access to publications explaining the development of IB1001.
 
STAY TUNED! We will announce the next topic in the new NTSAD webinar series to keep families and friends informed of timely topics.

Natural History Studies
 
Canavan Natural History Study and
Medical Record Retrieval
Aspa Therapeutics will soon be launching their comprehensive natural history study, CAN-Inform. The more data they collect from families, the better for the trial, and other future treatment trials. They ask for Canavan families who have or have had a child with the disease to participate. It is enormously helpful to have several years of data for a child so we hope all families - younger and older - will participate.
 
To learn more about record retrieval and participation, please email Veristat This email address is being protected from spambots. You need JavaScript enabled to view it..
 
GM1 Natural History Study 
University of Pennsylvania and Passage Bio are sponsoring a new natural history study focused on infantile and juvenile GM1 Gangliosidosis.
 
The data gleaned from the study will inform future trials and help to determine clinical endpoints that will show if a therapy is working.
 
The study is not yet recruiting but to learn more about the two sites and the contact information in the U.S. and the U.K. visit here.
 
 
 
GM1 Family Meeting, September 20, 2019 | Holiday Inn Bayside, San Diego, CA
 
NTSAD is sponsoring the GM1 Family Research Meeting held in San Diego featuring several speakers from other patient organizations, industry and research sites. It's been an exciting year to say the least and we're happy to be a part of it. NTSAD's Director of Family Services, Diana Pangonis, looks forward to meeting GM1 families in attendance and well as supporting the good work of the Cure GM1 Foundation. Learn more here about the GM1 Meeting.

 

In this issue

Family Support

New School Year: A Tough Milestone

Caring for Yourself

Caregiver Respite Program

Research

IntraBio webinar

Canavan and GM1 Natural History

GM1 Family Meeting in San Diego

Meet NTSAD's Board President

Staci Kallish, DO

NTSAD Events

Ninth Annual Day of Hope

Imagine & Believe

Remembrance

Say Their Names

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