In case you missed uniQure announcement regarding AMT-130, I’m posting the great news for the Huntington’s Disease community around the world. One of the locations for a clinical for AMT-130 is Ohio State.
~ Milestone Marks the First-in-Human AAV Gene Therapy Trial for Huntington’s Disease ~ Lexington, MA and Amsterdam, the Netherlands, June 19, 2020 —
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first two patients in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease have been treated. To read the complete news release, visit: AMT-130_Huntingtons_Disease_FINAL.pdf
I’m a Huntington’s disease advocate, the Chair for HDSA San Francisco Bay Area Affiliate, a blogger and the author of the nonfiction book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s.
John and I have donated 100% of the profit from the book to the nonprofit, Huntington’s Disease Society of America (HDSA). To date, we’ve gifted over $15,000.00. Review by Goodreads: Goodreads.com-Watching-Their-Dance
My family wants to see the eradication of this horrific disease that has devastated the Marin Family for generations.
On July 3rd, 2020, ABC News’ Linsey Davis follows two families grappling with the rare genetic disorder Huntington’s disease (HD), compared to ALS, Parkinson’s disease and Alzheimer’s disease all in one. (if video didn’t transfer, click here to watch the video: https://youtu.be/USwMplVl5vQ)
I was ecstatic when I saw the video on ABC. I don’t know how this came about but I bet the nonprofit, Huntington’s Disease Society of America (HDSA) had something to do with it!
Thank you ABC News.
The more awareness created about HD, especially by news organization like ABC, the more people will know about this horrific genetic disease with NO CURE. What-is-Huntington’s Disease?
HDSA and many other nonprofits are being challenged due to COVID-19. Most of HDSA fundraising events were changed to VIRTUAL and with so much uncertainty in our lives, donations are down. To make a donation to HDSA and help in the fight against Huntington’s disease, click here HDSA-Make a Donation
Author Therese Crutcher-Marin Book Signing in Auburn CA
Thank you for reading my blog. I’m an HD advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author of the nonfiction book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. 100% of the profits are donated to HDSA.
I’m posting a message from Jackie MacMullan, ESPN columnist and television analyst, and a supporter of the nonprofit, Huntington’s Disease Society of America (HDSA) for many years.
Author Therese Crutcher-Marin
Jackie has published many books in her career, so as a fellow writer, I’m sending her a copy of my nonfiction book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s, hoping she might mention the book so more dollars can be generated from sales that I will donate to HDSA. It’s a long shot! You never know!
From Jackie: “Nearly 30 years ago, I was working in the newsroom of the Boston Globe as a sportswriter when I fielded a phone call from a woman named Mae Long. She was the executive director of the New England chapter of the Huntington’s Disease Society of America and she wanted to know if I would shoot free throws to help find a cure for the disease. Article from: https://hdsa.org/living-with-hd/faces-of-hd/meet-jackie/
I was 22 years old at the time, a recent graduate of the University of New Hampshire, where I had played four years of basketball. Shoot free throws? Sure. But what was HD? I had never heard of it. What caused it? What were the symptoms? It was a mystery to me.
I went to Mae’s hoop-a-thon, and I was in for a rude awakening. I met a number of HD patients, some whose symptoms had not yet manifested themselves, and others who were in a wheel chair, unable to speak, in the advanced stages of a disease that I learned had no cure and indiscriminately decimated families.
It was impossible to forget those patients, their families and their poignant stories. Their plight stayed with me for days, weeks, and years. Before long, I was attending events, more hoop-a-thons, even spending a year as a national spokeswoman.
Three decades later, I remain committed to the HD community and its quest to find a cure. I’ve met and lost friends with HD, watching them slip away over time with uncommon grace and dignity.
Our family has befriended Meghan Sullivan, one of the rare patients who has been afflicted with early onset of HD. Meghan is only in her 20’s, but she is already in advanced stages of the disease. That has not stopped her and her mother Cheryl from being vocal advocates for the HD community.
I will never forget the evening Meghan stood in our kitchen and calmly explained to my husband Michael and me that she planned to put a face to HD for as long as her body and mind would allow it. She had already lost her father to the disease and promised him she would raise awareness. Her courage was both heartbreaking and inspiring. Though Meghan’s symptoms have limited her, she is still fighting, still honoring her commitment to her father.
The HDSA San Jose Team Hope 10K Run & 5K Walkis usually held at Campbell Park on the Los Gatos Creek Trail, but due to the Coronavirus, the event was changed to a VIRTUAL fundraiser.
FUNDRAISE FOR PRIZES!
Due to the economic impact of COVID-19, we anticipate raising most of our funds through peer-to-peer fundraising. And we want to reward you for your hard work and effort!
Local RAFFLE: (gift cards will be mailed)
Photo by alykat on Foter.com / CC BY
RAISE $500 & YOU’LL BE ENTERED INTO A RAFFLE FOR:
$100 Gift Card for “The Table” restaurant in Willow Glen
$100 Gift Certificate from “Sprouts Farmers Market”
MORE REASONS TO RUN & WALK
To help support and reward your amazing fundraising achievement to support the HDSA mission, HDSA is launching new Incentives!
$100+: HDSA Team Hope T-Shirt
$125+: HDSA Family is Everything Brandanna (facecovering)
$500+: HDSA Team Hope Contigo Water Bottle
$1,000+: HDSA Portable Cooler Totebag
$2000+: HDSA Stadium Blanket
The T-shirts and Face coverings will be shipped on a regular basis as the fundraising goals are met. The water bottle, cooler and stadium blanket will be shipped in December for fundraising achievements through November 30, 2020.
It is May 31st and we close out Huntington’s Disease Awareness Month #LetsTalkAboutHD
Author Therese Crutcher-Marin
For me, it’s not the end of raising awareness because I’m constantly posting Huntington’s disease (HD) information on all my social media platforms and wearing my Team Hope Walk T-shirt whenever I can. What is Huntington’s Disease
Since Huntington’s Disease is a rare disease, you may not know there are over 7,000 rare diseases. A rare disorder is a disease or condition that affects fewer than 200,000 Americans. On the NORD, National Organization for Rare Disorders, website Huntington’s Disease is listed. https://rarediseases.org/?s=H&post_type=rare-diseases
NORD’s mission statement: “NORD, a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.”
Wikipedia says, “The US organisation Global Genes has estimated that more than 300 million people worldwide are living with one of the approximately 7,000 diseases they define as “rare” in the United States.”
We Can Never Lose HOPE…….
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May is Huntington’s Disease Awareness Month. #LetsTalkAboutHD
In 2016, I asked Fran, my daughter-in-law, and Vanessa, my daughter, to run in the New York City Marathon on the Huntington’s Disease Society of America (HDSA) TEAM. New York Road Runners offers blocks of guaranteed, non-complimentary entries to qualified and selected nonprofits through the Official Charity Partner Program of the TCS New York City Marathon. https://www.nyrr.org/getinvolved/charity-partners/charity-partners-tcsnycm
Both of these women run marathons, 10K’s and half marathons so they were excited to apply and make money to help in the fight against the cruelest disease on the planet, Huntington’s disease.
The nonprofit, HDSA, participates in the TCS New York City Marathon Charity Partner Program. Fran and Vanessa had to apply to HDSA and commit to raising $3,500.00. Approximately 20 people were admitted to run on the HDSA Team and the team raised over $100,000.00.
Approximately 50,0000 people run the marathon. The event was very organized and the city was packed with bystanders. As they ran through the New York boroughs, people where partying and screaming and clapping for the runners. These two woman cut 10 minutes off their Marathon time because of all the support as they ran.
John, myself, Keith, Scott, Dawn and Jim Garret flew to NY and we all cheered them on. It was a blast and both Fran and Vanessa raised together $8,000.00
If you live in the San Francisco Bay Area, come to Lagunitas Brewing Company in Petaluma on February 3rd and help in the fight against Huntington’s disease. Lagunitas Brewing Company Address: 1280 N. McDowell Blvd. https://lagunitas.com/taproom/petaluma
A $20 entry fee, that includes a beverage, will get you in and you can wind down from your Monday while listening and/or dancing to the popular Sonoma County band, Poyntlyss Sistars Rockin’ Show Band.
Every year, Huntington’s Disease Society of America forms a team to run in the NYC Marathon as a fundraising event. The event is usually around the first of November. The pictures above are from the November 6, 2019 Marathon.
Our daughter, Vanessa, and daughter-in-law, Fran, ran in the 2016 NYC Marathon. Our family traveled to NYC to cheer them on. It was an incredible experience, so festive, fun and positive. Vanessa and Fran were ecstatic because they cut 10 minutes off their marathon time. Thousands of people cheered them on through the boroughs of New York.