aTyr Pharma releases results of early-onset FSHD trial

SAN DIEGO, April 24, 2017 (GLOBE NEWSWIRE) — aTyr Pharma, Inc. (Nasdaq:LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, today announced promising clinical results from its Phase 1b/2 003 trial assessing the safety and potential activity of Resolaris™ in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

Read the full press release here.

Help us promote World FSHD Day with a selfie video!

JUNE 20th is WORLD FSHD DAY!

World FSHD Day


Help us raise public awareness of FSH muscular dystrophy by making a video selfie, urging everyone to take part in this year’s World FSHD Day awareness campaign. Orange Slice SmileRaisingAware

Orange is the international color of World FSHD Day – so we’re using an orange slice to spread the word. All you need to do is make a short video of yourself sharing the message using the script below. Feel free to adapt the script to your own style and voice. Try to get good audio quality, then send your video by DropBox to June.Kinoshita@fshsociety.org. Continue reading

Intriguing research on tyrosine kinase inhibition as a potential therapy for FSHD: Sunitinib rescues muscle cells’ ability to develop

Written by Jim Albert
Eldersburg, Maryland

 

A cancer drug has been shown to potentially rescue some of the damaging effects of DUX4, the gene implicated in FSH muscular dystrophy. The laboratory of Peter Zammit, PhD, Randall Division of Cell and Molecular Biophysics, King’s College London, United Kingdom, in collaboration with Robert Knight, PhD, of the Department of Craniofacial Development and Stem Cell Biology at King’s, has published the results of its research on the activity of an FDA-approved drug, sunitinib, as having potential therapeutic activity for FSH muscular dystrophy (FSHD). Continue reading

Our Manhattan Project

Matthew Harms MD

The FSH Society’s March 11, 2017, meeting in Manhattan was a milestone in our efforts to nurture a network of stakeholders in one of our most concentrated urban centers. Hosted by FSH Society Board member Stuart Lai, this meeting featured two stellar speakers, Matthew Harms, MD PhD, of Columbia University College of Medicine, and Jia-Ray Yu, PhD, a postdoctoral fellow working in the world-renowned laboratory of Danny Reinberg, of the Howard Hughes Medical Institute at New York University Langone School of Medicine.

Two dozen patients and family members from New York City and beyond attended. For many, it was their first time meeting others with FSH muscular dystrophy. Continue reading

Double Your Donation… Easily and Free!

Corporate Matching Gifts: Does Your Company Match Your Donation?

Did you know many companies offer a matching gift program to encourage philanthropy among their employees?  And that some companies will even match to spouses, retirees, and board members?

By simply completing the matching gift form from your employer, you may be able to double, or even triple, the impact of your gift!

Click the button below to find out if your company will match your gift:  matching-gifts-red

If you do not find your employer listed, please check with your company’s Human Resources department to see if your company offers a matching gift program. Continue reading

FSH Society Awards $541,133 in Funding for FSH Muscular Dystrophy Research

Boston-based Non-profit Awards New Grants to Facilitate Search for a Cure

BOSTON – April 5, 2017 – Today the FSH Society, a world leader in combating facioscapulohumeral muscular dystrophy (FSHD), announced it has committed $541,133 in funding to five research projects that aim to break new ground in the search for a treatment and cure for FSHD. These grants follow the Society’s record breaking $1.36 million awarded in total research funding in 2016.

“These grants are a testament to the dedication of researchers within the FSHD community committed to understanding and solving how FSHD works through high quality peer-reviewed research” said Daniel Perez, President and CEO of the FSH Society. “With these grants we look to build upon our record-breaking success in 2016, which would not have been possible without the generosity and sustained support of donors, Society management and staff, our Board members and volunteers.”

The following proposals submitted in August 2016 were approved: Continue reading

Win a Horizon Off-Road Electric Trike: Bid Now!

The revolutionary Outrider Horizon Trike could be yours!
Bids accepted until 12 noon ET April 11th, 2017

We are auctioning off a Horizon Off-road Electric Trike – model 210A, an adaptive, all-terrain electric trike that can be customized for riders of all abilities. This revolutionary Horizon Trike will be shipped to you right off the production floor, and can be customized to your specifications. Check it out in the video above! Only a limited number are in production so the only way you can get your hands on the extraordinary Horizon is to bid on it by April 11th, 2017!

ElectricBike.com says “This counts as my favorite test ride of any production electric bike ever…Outrider trikes are so much fun to ride, that anyone spending a day on one in the right terrain will probably not forget that day for the rest of their life.”

The Trike was donated by one of the initial backers of the trike’s successful Kickstarter campaign, so 100 percent of this sale will go toward supporting the FSH Society’s mission! Continue reading

Update on Clinical Trial A083-02: a Phase 2 Clinical Study of ACE-083 in FSHD

The FSH Society has a long history of partnering with biotech and pharmaceutical companies to facilitate recruitment of patients and families for focus groups, provide patient input to clinical outcome measures, and participation in clinical trials. The Society also assists companies by providing connections, insights and scientific information in the research, therapeutic and clinical areas. For the ongoing trial of ACE-083, the FSH Society has worked with the drug’s developer, Acceleron Pharma, to better understand how FSHD affects patients through a survey (see story here) as well as to educate patients about the process of enrolling in the clinical trial. In response to the high degree of interest in the ACE-083 trial, Acceleron has worked with the FSH Society to provide the following update and FAQ. We thank Acceleron for the company’s commitment to patient education. Continue reading

FSH Society Talk Radio launches first episode

FSH Society Talk Radio host Tim Hollenback

We have launched a new service for our members, an internet radio show that will feature guest interviews and live on-air Q&A with our listeners. Hosted by Tim Hollenback, the show will stream live over BlogTalkRadio on the last Wednesday of every month, at 9:00 pm EST / 8:00 pm CST.Listeners can call into the live shows to ask questions and join a chat session with other listeners for real-time socializing, networking, and support. Episodes will be recorded and available as podcasts after the shows.

Listen to our pilot episode, an interview with FSH Society executive director June Kinoshita, who answers questions about current research, clinical trials, and the FSH Society’s role in supporting and educating the FSHD community.

FSHD patient survey results presented at MDA Conference

At the Muscular Dystrophy Association’s biennial scientific conference, held in Washington, DC, on March 19-22, 2017, researchers from Acceleron Pharma presented a poster about the most prominent symptoms and daily life impact of FSH muscular dystrophy, as reported by patients and caregivers. The report was based on results from a survey developed by Acceleron in collaboration with Dr. Jeffrey Statland of the University of Kansas Medical Center and June Kinoshita from the FSH Society. Researchers at aTyr Pharma also contributed comments on the survey design. Continue reading