Holiday Tea for a Cure

You’re Invited!

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This event is a fun-filled family holiday tea at a beautiful historic location!  The building was designed by Julia Morgan and is located on the UC Berkeley Campus.  We used to always take Iris to San Francisco for a holiday tea, but that stopped when she had to start a special diet due to her condition.

This is our very first holiday tea!  The event is open to young and old and we strongly encourage families to attend.  We will have a wonderful brunch and many fun activities:

  • Pictures with Santa
  • Story time with Santa
  • Silent Auction
  • Face painting
  • Balloons and Elves

The menu is as follows:

  • Assorted Finger Sandwiches (pb&j, cucumber, etc.)
  • House-Made Scones with Preserves
  • Roasted Vegetable Frittata with Mozzarella Cheese
  • Artisan Bakery Cinnamon Raisin French Toast with Warm Maple Syrup
  • Crispy Hash Brown Potatoes
  • Fried Chicken Tenders
  • Traditional Mac N’ Cheese
  • Pigs in a Blanket
  • Apple wood Smoked Bacon
  • Sliced Seasonal Fruit

Desserts:

  • Vanilla Cream Puffs
  • Cheesecake Bites
  • Brownie Bites
  • Cookies
  • Mini Crème Fraiche Panna Cotta with Strawberries

Coffee and Decaffeinated Coffee, Select Teas, Assorted Chilled Juices
The Cure GM1 Foundation was founded by Christine Waggoner and Douglas Dooley.  This 501(c)(3) was created in an effort to save their sweet daughter Iris’ life and to save the lives of all those affected by GM1 Gangliosidosis.  GM1 is a rare and fatal neurological disease that primarily affects babies and children.  Some might describe the condition as a pediatric disease with elements of Alzheimer’s and ALS/Lou Gehrig’s disease.

100% of the net proceeeds will be donated directly to the Cure GM1 Foundation to help fund a clinical trial for GM1 Gangliosidosis.  We hope with all our hearts that Iris will be eligible for the treatment and that many other children affected by GM1 will also be treated.

A donation in excess of the fair market value of $60 per person for the cost of the food, event, and entertainment is tax deductible.

Cure GM1 is also seeking sponsors for the event:

$2500 Gold Sponsor

  • Social Media Recognition
  • Event Sponsor Banner and Posters
  • Web site Recognition

 

$1250 Silver Sponsor

  • Social Media Recognition
  • Event Poster Recognition

 

$500 Bronze Sponsor

  • Event Poster Recognition

For more information on our nonprofit and about Iris, please see:

http://www.curegm1.org

http://www.sweetiris.org

October 2016 Cure GM1 Update

What an Amazing Month!

The Cure GM1 Tongue Twister Challenge was launched and we’ve had an amazing response from many friends and supporters spreading awareness.    Please see the video and page about the challenge.  The challenge is going on in full force now.  Please join in! On our Facebook page, you can see a bunch of videos. Each video has its own unique story.  Amazingly, we can not keep up with all of the videos!

Here’s the page: Cure GM1 Tongue Twister Challenge

October Fundraising Update – Incredible!

Truly, September and October so far have been the best months of 2016 for our small and mighty nonprofit.  We are truly grateful to the Heatherly and Bragg families and all GM1 families who have joined in by supporting our mission to cure GM1 Gangliosidosis.  This is the worst possible way to make new friends, i.e., we wish no one had to suffer from this awful condition.  Still, there are some truly wonderful people who are now part of our Cure GM1 community whom we would never have met otherwise.

We’re united in this fight for a cure!  Thank you to all the amazing donors and supporters for making September a HUGE month with $128,000 in donations!  There are also many pending corporate matches that will roll in very soon.  The full proceeds from events will roll in as well.

Prayers for Porter

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Prayers for Porter held a beautiful event for Porter’s 4th Birthday in Alabama.  This was the Heatherly family’s second annual birthday fundraiser.  A ton of press also featured Porter’s story.  How wonderful to see continued support this year!

A Cure for Clara

A Cure for Clara is in full swing.  Little sweet Clara was diagnosed in September 2016 with late infantile GM1.  Her family and parents jumped right into action and they are truly helping to advance our gene therapy program with an incredible fundraising campaign.

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See A Cure for Clara here! https://www.crowdrise.com/cure-gm1-for-clara

Upcoming Fundraisers

Run to Help Cure GM1

Participate Virtually, Purchase a Running Shirt, Sign Up!

No order minimums for this t-shirt campaign! Join us for the Berkeley Half Marathon! Buy a shirt to help us raise money to get GM1 Gangliosidosis gene therapy to clinical trials!
Multiple male and female styles available!
T-shirts: https://represent.com/run-represent-hope-help-cure-gm1

To Donate, to join the team,  and to find more information about the race team, check out CrowdRise! https://www.crowdrise.com/CureGM1BerkeleyHalfTeam

To participate virtually, just join the Crowd Rise campaign, choose a path to run or walk and invite your friends to support you!

The Berkeley Half Marathon will be taking place on November 20 and some awesome runners will be using it as an opportunity to fundraise for this amazing cause to help save children’s lives.

Family Holiday Tea at the Berkeley City Club 12/18 11:30am-2:30pm

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If you are in Northern CA, we have two upcoming events here locally in the East Bay!

Save the date for our first ever Family Holiday Tea!   We want to emphasize that children are invited and this is a family friendly event. The tea will take place at the Berkeley City Club and it’s a family event with Santa, music, delicious food, face painting, balloons, and a silent auction.  We’ll send out the invitations soon very shortly for early birds!

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Sponsors Needed

If you are interested in being a sponsor for the event or if you know someone who might be willing to be a sponsor, please let us know.

Gene Therapy Update

A short delay in actually sending the first document called the Pre-IND has occurred.   The hope is to get the Pre-IND really, really, truly in the mail in November!  Fingers crossed!  We can not emphasize enough how truly complicated and convoluted this process is for all parties involved.

We are sharing this information openly and honestly because we feel our donors, supporters, and families deserve to understand the truth.

The project is still on track for 2017, but there are many, many steps and pieces that will need to fall into place to meet our goal.

As you know, one of the very most important pieces is funding.  We are still fundraising because the cure is not here.   Those affected by GM1 experience the full brunt of there being no treatment.   The funds needed now are for manufacturing and running the clinical trial.  The budget is very lean and this is currently a tight situation financially.  We’d like to change that and to be able to have more flexibility.

To make a trial happen and to navigate all the complexity, we still need your support.

This project is for the world and for the entire GM1 community.  Frankly, it’s also for all humanity and could advance medicine.  Please make a contribution today to this incredibly worthy cause!

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Cure GM1 Tongue Twister Challenge

See the video and instructions below!  Together we can make a difference!

How do I do it?

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More Detailed Instructions

  1. MAKE A VIDEO of yourself, your family, or a group of friends saying “GM1 Gangliosidosis”  5 times as fast as you can!
    • Print and hold up a TEXTBOARD: #GM1TongueTwister in your image or video that says “Donate at http://www.curegm1.org”   Download an textboard HERE.
    • DON’T FORGET to say “I am doing the #GM1TongueTwister challenge to help save the lives of children with GM1 Gangliosidosis.  Donate at http://www.curegm1.org.”  
    • ADD your own creative flare to add your own personal touch and humor to your video!
  2. NOMINATE three or more other people to take part
  3. REMEMBER – If you laugh or smile you have to donate!
  4. POST IT to Twitter, Facebook or Instagram using the hashtag #GM1TongueTwister
  5. DONATE at https://curegm1foundation.org/donate/ or our generosity.com crowdfunding page: https://igg.me/at/-azYuaOSnck

Here’s a PDF file you can print to hold up in your video!

  1. Click on this file link and print out the file! gm1TongueTwister
  2. Hold up the print at the end of your video!

Who are you helping?

All the funds are being put towards medical research to save dying children’s lives!  More specifically, we’re in the final stages of funding the first ever clinical trial for a cure for GM1.   We need to make it through toxicology and manufacturing of the treatment.  This is VERY close, but we need your help to help make a cure a reality!

Donations are 100% tax deductible.  There are no paid employees at the Cure GM1 Foundation and the maximum amount possible other than credit card billing fees is being put directly towards a cure.  

Cure GM1 September 2016 Update

Read the latest updates from the Cure GM1 Foundation below!

Porter’s 4th Birthday Fundraiser in Auburn, Alabama

The Heatherly family have been such amazing advocates for their son Porter who has infantile GM1.  We are so truly grateful for their support to help cure GM1 Gangliosidosis and the intravenous gene therapy program.   As with all children who are affected by GM1, the disease continues to take a tremendous toll on this sweet boy.  Our thoughts are with the Heatherly family as always.

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If you are in the Auburn, Alabama area, Porter’s fundraiser will in on October 1st at the Auburn University Club.  Please see http://www.curegm1fundraiser.eventbrite.com  for more details.  They are looking for auction items and sponsors as well.

Join the Cure GM1 Task Force!

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A small team of GM1 dedicated and passionate parents currently composes the Cure GM1 Task Force.  The role of the task force is to provide a forum for parents to help the Cure GM1 Foundation and to help organize fundraising efforts.

The task force structure consists of monthly conference calls, a mailing list, and a private Facebook group.  There’s lot of brainstorms and members of the group lend their skills, support, and talents to each other and the foundation.

If you have great fundraising ideas or just want to help, we’re all ears!  If you are interested in joining,  please contact us.

Run to Help Cure GM1

Participate Virtually, Purchase a Running Shirt, Sign Up!

No order minimums for this t-shirt campaign! Join us for the Berkeley Half Marathon! Buy a shirt to help us raise money to get GM1 Gangliosidosis gene therapy to clinical trials!
Multiple male and female styles available!
T-shirts: https://represent.com/run-represent-hope-help-cure-gm1

To Donate, to join the team,  and to find more information about the race team, check out CrowdRise! https://www.crowdrise.com/CureGM1BerkeleyHalfTeam

The Berkeley Half Marathon will be taking place on November 20 and some awesome runners will be using it as an opportunity to fundraise for this amazing cause to help save children’s lives.

Holiday Tea for a Cure

Volunteer, Donate an Auction Item, Mark Your Calendar!

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On December 18th at the Berkeley City Club in California, Cure GM1 will be hosting a holiday tea and auction.  If you would like to contribute and auction item, please mail them to the foundation by December 1st and please contact us to let us know what you are sending.   Volunteers  are also needed.  To attend more details to follow!  It will be a lot of fun with Santa, entertainers, and delicious food in a beautiful historical location.

 

Meet Lenka From Poland

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Thousands and thousands of miles away in Poland, a beautiful, sweet girl named Lenka suffers from GM1 Type 2.  Lenka was born on August 18, 2004.   Lenka’s early childhood proceeded normally and her sociable and bold personality emerged.

At the age of five, Lenka first developed what appeared to be insignificant symptoms. She often stumbled and fell more often than other children her age.  A neurologist helped investigate the cause. The neurologist noticed a slight decrease in muscle tone in the legs and developmental coordination disorder called dyspraxia.   The neurologist reassured her parents and told them not to worry .  The doctor seemed unconcerned and commented that she might not become a ballerina.

Unfortunately, in spite of sensory integration therapy, hippotherapy, and swimming therapy,  at the age of 7, new disturbing symptoms emerged.  The array of symptoms included slurred speech, blurred drawings, stacking problems, leg pain, and problems with balance.  After many medical tests and procedures, doctors misdiagnosed Lenka with “dyspraxia of unknown etiology.”

Finally, in 2012,  Lenka’s parents continued their search for a true diagnosis, knowing in their hearts that something more insidious afflicted their sweet girl.   After years of searching,  Lenka was diagnosed with GM1 Gangliosidosis at the Children’s Health Center in Warsaw.

Unfortunately, Lenka’s diagnosis story bares many similarities to many other children affected by GM1. The correct diagnosis is truly hard to find and can take many years to obtain.  The prolonged time to diagnosis reduces the possibility of early intervention.  Furthermore, there is no treatment for Lenka or any other person afflicted by GM1 Gangliosidosis and all the interventions are only intended to increase quality of life.

The hope for Lenka and all those affected by this cruel disease is medical research.  As a result, Lenka’s family recently contributed a $5000 donation to the Cure GM1 Foundation to the gene therapy program.  The Cure GM1 Foundation thanks Lenka and her family for their generosity and support.  Together we can make a difference even when we are worlds apart.

 

Intravenous Gene Therapy Program Update

Sunset landscape

Sunset landscape

For more detail’s, please see last month’s post.  Rest assured work is proceeding!  The first Pre-IND (investigational new drug) meeting with the FDA will be scheduled before the end of the year.  The feedback from the FDA will determine the next steps in the research to send in the full IND application for the clinical trial.

If there was ever a window of time when we need your support, it is now!  Clinical trials can not be conducted without adequate financial resources. 

Please consider a donation now to help support the next steps to make the gene therapy trial a true reality.  These could potentially and hopefully be the first steps towards the end of GM1.

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Lenka’s Story of Hope – Lenka i Dobra Spółka

Thousands and thousands of miles away in Poland, a beautiful, sweet girl named Lenka suffers from GM1 Type 2.  Lenka was born on August 18, 2004.   Lenka’s early childhood proceeded normally and her sociable and bold personality emerged.

At the age of five, Lenka first developed what appeared to be insignificant symptoms. She often stumbled and fell more often than other children her age.  A neurologist helped investigate the cause. The neurologist noticed a slight decrease in muscle tone in the legs and developmental coordination disorder called dyspraxia.   The neurologist reassured her parents and told them not to worry .  The doctor seemed unconcerned and commented that she might not become a ballerina.

Unfortunately, in spite of sensory integration therapy, hippotherapy, and swimming therapy,  at the age of 7, new disturbing symptoms emerged.  The array of symptoms included slurred speech, blurred drawings, stacking problems, leg pain, and problems with balance.  After many medical tests and procedures, doctors misdiagnosed Lenka with “dyspraxia of unknown etiology.”

Finally, in 2012,  Lenka’s parents continued their search for a true diagnosis, knowing in their hearts that something more insidious afflicted their sweet girl.   After years of searching,  Lenka was diagnosed with GM1 Gangliosidosis at the Children’s Health Center in Warsaw.

Unfortunately, Lenka’s diagnosis story is not unusual for a child diagnosed with GM1. The correct diagnosis is truly hard to find and can take many years to obtain.  The prolonged time to diagnosis reduces the possibility of early intervention.  Furthermore, there is no treatment for Lenka or any other person afflicted by GM1 Gangliosidosis.  All the interventions are only intended to increase quality of life for comfort only.  The disease ravages the central nervous system and the body.

The hope for Lenka and all those affected by this cruel disease is medical research.  As a result, Lenka’s family recently contributed a $5000 donation to the Cure GM1 Foundation to the gene therapy program.  The Cure GM1 Foundation thanks Lenka and her family for their generosity and support.  Together we can make a difference even when we are worlds apart.

 

August 2016 Update – Big News!

Intravenous Gene Therapy Update

AMAZING news! We recently received a detailed update on the progress of the Auburn/UMass GM1 intravenous (IV) gene therapy research program. Work is truly active towards the creation of a clinical trial in human children! The details of the progress towards creating an IV gene therapy clinical trial in the United States are below.

The Cure GM1 Foundation is supporting development of IV gene therapy for GM1 gangliosidosis as follows:

A. 2 treated GM1 cats are currently 32 months old and doing well (untreated GM1 cats live 8 months). The treated cats behave and function normally. Their primary abnormality is weight loss.

B. The funds are supporting a regulatory consultant, who will inspect vector production facilities as needed and who is writing the pre-IND (investigational new drug) document for submission to the FDA. This is the first step in approval of a clinical trial, and the FDA has 2 months after submission to comment on the pre-IND document. The regulatory consultant will respond to the FDA’s comments by filing a formal IND application for final approval of a clinical trial.

C. Funds are supporting the initial steps of vector production, in which a test batch is made from highly purified DNA stocks.

D. A portion of the funds will be used to perform toxicity and bio distribution studies in normal mice, a step required by the FDA to demonstrate safety of the vector that will be used to treat children.

These are the 4 main uses of the funding so far. A and B are underway. We are waiting to begin production of the test batch in C. Studies in D can not begin until the precise method of production is determined.

The road to clinical trials is arduous. We are truly fortunate that decades of research has been conducted to arrive at this point. The exact timing of the clinical trial is still to be determined and the exact parameters of the clinical trial are also still to be determined.

We still hope the trial could be in 2017. The determination of the exact date depend on the regulatory requirements and the manufacturing schedule. Hopefully, as long as all the stars align, the trial will commence by the end of 2017.

Although we are very encouraged by this wonderful news, many steps remain and significant funding is still required for manufacturing and testing.  Continued support of this program and the Cure GM1 Foundation will help guarantee that this trial becomes a reality.

Children’s lives are at stake and this treatment was needed over 130 years ago when this category of diseases was first identified.

Please help support Cure GM1 on this incredibly important mission!

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News

A few other highlights from recent efforts to bring more attention to our tremendously worthy cause.

A Mother’s Original Song for a Cure

Click on the image to listen!

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Marilee Leishman created an incredibly beautiful and moving song that truly speaks for all those affected by GM1 Gangliosidosis.  Please click on the image above to learn more about the song.

Porter’s Annual Fundraiser and Upcoming 4th Birthday

All those affected by GM1 know how truly momentous birthdays are!  Porter’s family will be holding his 2nd annual birthday fundraiser on October 1st in Alabama.

Jessica Kelly, a GM1 researcher in Auburn is also running a marathon in honor of Porter and all those who suffer from GM1.  To learn more about the run or to purchase a t-shirt, please click the image below.

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More details to follow on Porter’s fundraiser!

Back to School T-Shirts for Iris

A friend and supporter created an original design to help support GM1 awareness and to contribute to research.  To learn more about this project, please click the image below.

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Run the Berkeley Marathon to Raise Awareness

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Some kind folks have expressed interested in running the Berkeley Half Marathon for our Sweet Iris and for a cure for GM1! The race is November 20th, 2016. To register and to learn more see below:
https://www.crowdrise.com/CureGM1BerkeleyHalfTeam
http://berkeleyhalfmarathon.com

If you are interested let us know. If you don’t live nearby, you can participate virtually! Just skip the Berkeley Half Marathon registration, run, and fundraise.  Please let us know if you would like to join!

Thank you!

Thank you from the bottom of our hearts for your support!  The progress made so far would not be possible without all those who have contributed so generously!  We are truly grateful.  Do not hesitate to contact us with any questions or concerns.  Thank you.

Sweet Angels Song for a Cure

Please watch and share this amazing music video and song by Marilee Leishman written for her two sons Eli and Evan who suffer from GM1 Gangliosidosis Type 2.

To Donate, please see this link on generosity.com: https://igg.me/at/O-Psh548uj0

To purchase the song,  here’s link:  Google Play Purchase.

 

 

Believe – I Am Rare Original Design

Help support a cure and those who suffer from GM1 Gangliosidosis today by declaring that you are rare!

The Cure GM1 Foundation is honored to release a new t-shirt design in collaboration with a friend and supporter who specializes in designs using maps.  This is a playful map design available in infant, children, and adult sizes.

Two possible variations are available and many, many different styles.  Adult male and female clothing, kids shirts, onesies, sweatshirts, and stickers are all available in 2 color schemes.

  1. Black text for printing on light colored shirts
  2. White text for printing on dark colored shirts

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PURCHASE and support a cure today via these links:

1. Black text for use on light apparel:
2. White text for use on dark apparel:
These are just a few examples of the available products and styles:

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And stickers!

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Lots of different designs are available and all net proceeds will be directly donated to help cure GM1 Gangliosidosis.

Please share this fun design and don’t miss out on this unique and fun design!

Cure GM1 Foundation Family Liaison

The Cure GM1 Foundation welcomes Dee King in the role of Family Liaison.  Dee’s daughter Fiona passed away on December 23, 2015, four and a half years after the devastating diagnosis of infantile GM1.   Dee has graciously volunteered for Cure GM1 to help support other GM1 families coming to terms with this disease.

Dee’s grace and support of the GM1 community is truly exemplary.   Cure GM1 is honored to welcome her to this new role to better support the Cure GM1 community.

 

To contact Dee, please look her up on Facebook or Contact Us for her phone number and e-mail address.

Cure GM1 Parent Conference Call

 

Reminder that we have  a parent conference call tonight 5/16/16 at 6PM PST.

The conference call access and directions will be sent directly to those who contact us.

You can use the contact form to request the directions.