A Cure
Can't Wait

Are you a rare patient or caregiver with concerns related to the COVID-19 outbreak? Do you need strategies for coping, staying healthy, and reducing anxiety during this uncertain period? 

NORD (National Organization for Rare Disorders, Inc.) is hosting a special webinar on Tuesday, March 31, at 2:00 p.m. EDT to provide guidance on living with a rare disease and maintaining your physical and mental health in the time of COVID-19. This webinar is perfect for patients, caregivers, advocates, and others.

The webinar will feature Dr. Marshall Summar, Chief, Division of Genetics and Metabolism, and Director of the Rare Disease Institute at Children’s National Hospital; Dr. Bernhard Wiederman, Infectious Disease Specialist at Children’s National Hospital; and Dr. Albert Freedman, Counseling Psychologist and rare parent.

Register for the webinar at: https://globalmeetwebinar.webcasts.com/starthere.jsp?ei=1295204

The world is grinding to a halt because of Coronavirus. Except for one thing that continues at the same pace … time. And time is the greatest enemy to children with Sanfilippo Syndrome.

Coronavirus is impacting the Sanfilippo community on a few crucial and intertwined fronts:

The significant impact on the first two will undoubtedly delay aggressive progress to find a treatment or cure for this rapidly-degenerative disease, as well as experimental treatments for children. It’s happening already. The third adds increased worry and stress to parents already dealing with the worst imaginable diagnosis for their child.

Some of the ways that Coronavirus is impacting the Sanfilippo community:

• Planned fundraising events in the immediate days are being evaluated to possibly be postponed or cancelled. Just a few days ago, the Abby’s Alliance 5K in Houston, TX, was cancelled.
• Fundraising events in the coming weeks and months are in limbo as organizers wait to see the progress of the virus. Among those up in the air are the Super Eliza 5K in Columbia, SC, coming up in April, and other events in May and June.
• The plan to launch a new viral video campaign in late March, hoping to raise over $1M for Sanfilippo research, has been put on hold because the time is no longer right, based on Coronavirus in the news.
• In-person meetings with donors are being cancelled, postponed, or being conducted via other means.
• Research and clinical trials, including enrollment of the Foundation’s sponsored clinical trial treating children to reduce inflammation in the brain, are being put on hold as facilities restrict access and continued concerns about travel exposure.
• Meetings with regulators, such as the Foundation’s important upcoming meeting with the FDA, are being changed from in-person to virtual settings.
• Research projects to be funded later in 2020 and 2021 being re-evaluated based on ability to bring in the funding needed.
• Staff at institutions working on Sanfilippo, including the Foundation, are navigating the disruptions to their lives, such as state-mandated school closures, and how it impacts their ability to work.
• Children with Sanfilippo Syndrome may be at risk for more severe complications due to conroovirus, particularly those in the more severe stages of Sanfilippo Syndrome.
• One of the characteristics of Sanfilippo is that children tend to put all sorts of things in their mouth including their hands, which makes it more difficult to keep potential viruses away, particularly the younger more active children with Sanfilippo.
• For families who have children that take immunosuppressive medications (ie prednisone, tacrolimus, mycophenolate mofetil, and others), please consult with your doctor about any precautions needed for your individual circumstance. Information is quickly changing, so please keep updated on CDC website, and see helpful resources links below.

Like you, we wait to see how far and long the impact of Coronavirus goes. In the meantime, we will continue doing our best to keep the fight to cure Sanfilippo moving forward as fast as possible. We are also working on short term, and longer term impact strategies.

We believe that this pandemic has given many a new and “all too real” perspective on the stresses that come with worrying if your loved ones will remain healthy. It’s just a glimpse into what Sanfilippo parents face every day after diagnosis. We understand that you and your family’s health are the primary concern, as well as your finances. And these should be as these are two of our primary needs in life, and we are right there with you. We hope we ALL can get back to a “new normal” soon.

Through it all, we are incredibly humbled and genuinely thankful for you. Your support – past, present, and future – means everything to us in the ability to maintain progress against this disease. One of our taglines in the past has been “In It Together.” Well we are all truly “in this together” and we will get through it together.

We wish you and your families safety and health throughout these trying times. We truly appreciate you and thank you.

— Cure Sanfilippo Foundation

Resources

Information on Coronavirus

• Center for Disease Control (CDC): https://www.cdc.gov/coronavirus/2019-ncov/index.html
• HealthyChildren.org: https://healthychildren.org/English/health-issues/conditions/chest-lungs/Pages/2019-Novel-Coronavirus.aspx

Activities for Kids

There are so many free resources available now in light of the pandemic. Here are just a couple that we enjoyed during our previous period of isolation.

• BrainPOP: https://www.brainpop.com
• Khan Academy: https://www.khanacademy.org/

One Sanfilippo family’s experience relevant to today’s Coronavirus concerns about self isolation

Their story of isolation is unique and relevant at the moment as many people are wondering about voluntary and mandatory quarantines because of Coronavirus (COVID-19) and how to navigate living in isolation.

To help others thinking about isolation because of Coronavirus, to give some perspective, and to also raise awareness of Sanfilippo Syndrome, the O’Neills made themselves available to reporters interested in the family’s experience and strategies for managing work, school, children, etc. while in quarantine.

Their experience has been featured in:

• Today.com 
• ABCNews.com

More Information:

The O’Neills’ daughter Eliza has Sanfilippo Syndrome, a terminal, neurodegenerative disease like Alzheimer’s in children. Her only chance at a cure was participating in a clinical trial that uses a common virus to deliver the treatment. If a child has previously had the virus, they are ineligible for the clinical trial because their bodies would be immune.

There were no promises that she would get in the trial. But if she was exposed to virus before the trial happened, she would be instantly excluded. This was the O’Neills’ one shot at a chance for a different life for her. So they took the extraordinary measure of isolating themselves in their house until the clinical trial started and patients were enrolled to make sure she didn’t catch the virus beforehand.

They expected the isolation to last a couple of months. It turned into 726 days. From May 14, 2014, to May 10, 2016. During that time, Glenn worked from home, their son Beckham and Eliza attended school remotely, they continued specialized therapies for Eliza, they had food and supplies delivered to the house, and they made heavy use of technology to stay connected with family and friends. Glenn and Cara also operated Cure Sanfilippo Foundation and raised $2.1 million through a viral video (www.SavingEliza.com) to help fund research to cure Sanfilippo Syndrome, including the clinical trial.