The Most Precious Gift for Medical Research

by Facioscapulohumeral Society/FSH Society
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The Most Precious Gift for Medical Research
The Most Precious Gift for Medical Research
The Most Precious Gift for Medical Research
A Fulcrum researcher at work.
A Fulcrum researcher at work.

Your generous support of our program to deliver muscle tissue from FSH muscular dystrophy patients to researchers has led to the development of an innovative "muscle in a dish" that is now being used for drug discovery. According to a report in Clinical Leader, Massachusetts-based biotech company Fulcrum Therapeutics “is using tissue donated from patients with facioscapulohumeral (FSH) muscular find a treatment. The tissue is used to create research models that look and respond much like natural human tissues.”

The article goes on to explain, “Since Fulcrum opened for business in 2016, it has built a relationship with the FSH Society, a patient advocacy group championing FSH drug development and clinical trials, as well as funding for research. The group has been critical in helping Fulcrum understand the patient community and develop clinical outcome targets that are meaningful to both patients and families.”

“In addition to helping Fulcrum connect with the patient community, the FSH Society has also worked to draft a tissue protocol for patient donations through the NDRI (National Disease Research Interchange). The FSH Society partnered with the NDRI to create a nationwide registry of individuals with FSH which has been integral in allowing the NDRI collaboration to occur.”

The article explains why primary muscle tissue donated by patients is critical for Fulcrum’s ability to develop a model system for drug testing.

We thank you sincerely for making this breakthrough possible through your support of our tissue donation program.

Read the full article: Tissue Modeling May Cut Clinical Trial Time


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With your help, we'll help kids like Keisha
With your help, we'll help kids like Keisha

Our national FSHD biospecimen registry is up and running. We have successfully completed a surgical and post mortem donation, with precious FSHD tissues going directly to a cutting-edge laboratory. What we now need to do is greatly increase the number of individuals who have signed up for the registry. Every time I'm in front of an audience of patients and families, I urge them to register now, just the way many of us sign up to be an organ donor when we receive our drivers' license. This is not something to put off to the eleventh hour. Whenever I hear news of an FSH muscular dystrophy patient who has had surgery or who passed away without making a tissue donation, it feels like a big loss to the community. These tissues are critically important for advancing our knowledge about how to cure FSHD.

So, early next year, we will launch an educational campaign to raise awareness about the biospecimen registry. We will send out a questionnaire that will test people's knowledge about tissue donation, and hopefully by the time they complete the simple survey, they will understand and be motivated to register. If you are affected by FSHD, I hope you will make time this holiday season to sign up. Please encourage affected family members to do so as well. This is the most precious gift you can give to your future, your family's future, to the FSHD community as a whole. 

To register, please contact NDRI at 800-222-6374. Be sure to mention that you wish to sign up to the “FSHD registry” when you call.

With gratitude to all who support this project, and Happy Thanksgiving!


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Our work today will benefit kids like Madilyn
Our work today will benefit kids like Madilyn

To all of you who have supported our FSHD Tissue Registry project, we are deeply grateful! We continue to put significant effort into fine-tuning the registry system, with bi-weekly calls between Daniel Perez, CEO, President, and Chief Science Officer of the FSH Society, and the amazing staff at NDRI. Our tissue donation registry is now capable of recovering the most comprehensive collection of FSHD muscle samples ever attempted.

We’ve received strong interest from the scientific community. To date, we have registered six labs that are ready to receive post mortem tissue donations, four who wish to receive surgical donations, and two researchers who need tissue from unaffected family members.

Around two dozen patients have also registered as tissue donors. That’s a great start, but we need many more to sign up! Registering to donate tissue is very much like signing up to be an organ or bone marrow donor. If you know that this is something you wish to do, please make arrangements now because later… may be too late.

Over the past year, we have unfortunately lost a number of FSH Society members. Some had reached a ripe old age, while others were taken too young due to accidents or illness. Their families reached out to us during their loved ones’ final moments hoping to make a tissue donation, but by then it was impossible to obtain signed consent forms, obtain permission from the hospital, and locate a pathologist within the NDRI network who could recover the tissue.

Considering the rarity of FSHD—and how rarer still it is for a patient to pass away or to undergo surgery—these lost opportunities are painful. Registering in advance is the key to fulfilling patients’ wishes and ensuring that these most precious gifts for FSHD research can go forward. What we do today will build a brighter future for everyone with FSHD.

What can you do? If you are affected yourself, be sure to register with NDRI now. If you have family members or friends who are affected, speak with them about the importance of registering soon. To register, please contact NDRI at 800-222-6374. Be sure to mention that you wish to sign up to the “FSHD registry” when you call.

Thank you so much for your help.


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Maureen's son Liam presenting on FSHD science
Maureen's son Liam presenting on FSHD science

In November of 2016 I underwent Scapula Fusion surgery in Baltimore, MD.  During that surgery I had the privilege to donate muscle tissue for FSH research.

The donation itself was relatively simple. The paperwork prior to the surgery was minimal and was mainly taken care of by the doctor’s office.  The procedure itself was included as part of my overall surgery, and did not cause any further pain or loss of mobility.  

Several months prior to the surgery June Kinoshita from the FSH Society reached out to me asking if I would be interested in donating muscle tissue taken from my surgery.  It wasn’t even a question in my mind. Without hesitation I committed to donating muscle. 

I was diagnosed with FSHD in 2011. My son Liam, then only 6 years old, was also symptomatically diagnosed.  Since that time I, like everyone else I have spoken to with FSHD, have been on a quest to learn everything I can about the disease and most importantly how to treat it, how to slow the symptoms, how to cure it. 

As a mother, I have not encountered any obstacle as frustrating and as upsetting as bringing my son to doctor after doctor, only to be told there is no treatment, there is no cure.  My only choice is to sit idly by and watch and wait for progression, and pray that the wonderful doctors and scientists come up with a treatment before his disease progresses. 

This is why I would not even call it a decision to donate, it was simply something I could finally do to be able to contribute to my son’s health.  In the past I have donated money which I know is of the utmost importance for the continuation of research.

Being able to donate tissue is something completely different.  It was finally something positive I could provide from having this disease. The knowledge that perhaps the tissue from my body will help a child, any child not lose their smile, or not lose the ability to raise their arms, or keep them walking fills me with such hope and gratitude. 

This disease can take away so many physical things, but it can also make us stronger in ways we didn’t know was possible. Helping others by donating tissue is a way to find strength in the midst of weakness. It is providing hope to our community.

Visit the FSH Society website for information on joining the FSHD Tissue Donation Registry.

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Researchers at Fulcrum Therapeutics.
Researchers at Fulcrum Therapeutics.

We have exciting news! Your donations have funded the first recovery of FSH muscular dystrophy tissue from a patient who had surgery last November. Among the labs that received the tissue is Fulcrum Therapeutics, a Cambridge, Massachusetts biotech that is working on treatments for FSHD. Using this muscle tissue, Fulcrum has developed FSHD primary muscle stem cells known as myoblasts which are capable of being expanded and cultured in their laboratories for many uses. Fulcrum will use these FSHD myoblasts for disease characterization and early drug discovery activities. The company would like to make these cell lines available for wider distribution to other researchers. 

We now have nearly a dozen research labs that have applied to receive tissues through this registry, and many patients who have registered to donate tissue. With your continued support, we will be able to deliver their invaluable donated tissues to the world's leading FSHD researchers. Thank you!

You can read more about Fulcrum's work here.


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Organization Information

Facioscapulohumeral Society/FSH Society

Location: Lexington, MA - USA
Facebook: Facebook Page
Twitter: @FSHSociety
June Kinoshita
Project Leader:
June Kinoshita
Lexington, MA United States

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