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FSH Watch 2016 Issue 1 is out!

2016FSHWatchIssue1_coverHot off the press, our current issue of FSH Watch keeps you up to date on important advances in research and medical care, as well as news about your Society, regional meetings, and upcoming events. Individuals living with FSHD share their thoughts about “coming out” and their responsibilities as advocates and research participants. Highlights include:

  • Human FSHD muscles cells have been successfully grown in a mouse, providing a possible new animal model (page 1);
  • First-ever World FSHD Day was celebrated on June 20 (page 1);
  • Dr. Rabi Tawil answers patients’ questions about the evidence-based FSHD care guideline published in 2015 (page 3);
  • A patient reflects on the responsibility that comes with volunteering for a clinical study (page 5);
  • NIH funding for FSHD research hits a new record (page 4);
  • EIM: An inexpensive, fast tool for clinical trials? (page 6);
  • Feed you soul. Artists with FSDH get extra creative (page 8);
  • Newly awarded FSH Society grants (page 16);
  • Upcoming patient meetings and events (page 20);
  • Progress in biomarkers for FSHD (page 22).

To read, download FSH Watch 2016 Issue 1.

2016 FSHD Connect Conference

Westin copley bostonRegister at the following links:

This year, for the first time, we have combined our two signature meetings into FSHD Connect, a single, three-day super-conference. Our events begin on November 10-11 with Research Connect (formerly the International Research Consortium Workshop). Open to active research professionals from around the world, the workshop is the premiere annual platform for clinicians, medical researchers and basic scientists to present and discuss new developments in FSH muscular dystrophy research. Continue reading

New member of our team

Beth Johnston

Beth Johnston

Beth Johnston has joined the FSH Society in August of 2016 as Development Officer. She will work with the Executive Director and the leadership team to create, execute and grow the Society’s fundraising plan and ongoing efforts.

Beth’s long history with the Society includes fundraising, Board membership, and grant writing. She will continue her commitment to raising awareness of both the disease itself and the function of the Society in this new development role. Continue reading

New FSHD biotech launched with $55 million

Screen Shot 2016-07-19 at 9.10.35 AM

From BusinessWire

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Third Rock Ventures, LLC today announced the launch of Fulcrum Therapeutics, a company focused on unlocking gene control mechanisms to develop small molecule therapies. Fulcrum will discover and develop small molecules that modulate the on/off control mechanisms that regulate genes.

Fulcrum was established with $55 million in Series A financing to further develop a cross-disciplinary product engine to identify and modulate gene regulatory targets core to disease. Through its product engine, Fulcrum will develop a robust pipeline across therapeutic areas, spearheaded by two initial programs in genetic diseases. The company is led by President and Chief Executive Officer Robert J. Gould, Ph.D., former President and CEO of Epizyme, and a long-time leader in drug discovery and development.
Continue reading

Help put FSHD on TV!

FSH Society public service announcements are available for television broadcast, but we need your help to get stations to actually air them. When stations hear from local viewers, they can be persuaded to take a closer look. With just an hour or two or your time, you can make a huge difference!

It’s simple: Just download the FSH Society PSA Toolkit, which tells you how to reach out to stations and provides a template letter/email to send to stations’s PSA or program directors.

Lake Party Thank You Party – Matching Gift Challenge!

2016_LakePartyThis year marks the final year that Michelle and Dave Mackay will have the Lake Party at their Michigan home, as it has been sold. They will host a thank you party at the Gull Lake Country Club this August 6 for friends who have supported the FSH Society through this event over the past three years. Please join us in thanking the Mackays for their extraordinary leadership in the FSHD community by making a gift in honor of their Lake Party. The Mackays will match every gift, dollar for dollar!!

UPDATE: The Mackays have upped the ante and offered a 2-for-1 match on any gift from all first-time donors to the FSH Society, up to a cumulative $50,000!! For those of you who have never donated to the Society, this is your opportunity to make your money stretch a long way toward a cure! Every dollar you donate becomes $3 and our total goal of $50,000 becomes $150,000! And if you are a regular donor (for which we thank you sincerely!), your gift will still be matched dollar for dollar.

If we meet this challenge goal, we will be able to fully support an exciting project just recommended for funding by our Scientific Advisory Board, which will test CRISPR technology as a way to silence DUX4 in promising new mouse model of FSHD.

Continue reading

Mouse grows a human muscle

With a little zap, transplanted human cells flourish in a mouse

By June Kinoshita, FSH Society

The green color indicates the presence of human spectrin protein, and shows that most of the cells in this muscle taken from a mouse are of human origin.

The green color indicates the presence of human spectrin protein, and shows that most of the cells in this muscle taken from a mouse are of human origin.

It’s like something out of Dr. Frankenstein’s underground laboratory, but a University of Maryland Medical School team led by Robert Bloch, PhD, has successfully coaxed human muscle precursor cells to grow and form mature muscle in a mouse. Among the tricks used to accomplish this feat was the application of electric currents to the transplanted cells.

The study, published in the journal Skeletal Muscle, notes, “These studies were supported initially by a fellowship … from the FSH Society.”  Continue reading

Mini Border 2 Border ride to raise awareness

Bob and Wally the Wombat

Bob and Wally the Wombat

Bob Louden, organizer of the monthly Western Washington patient support group, had to cancel his planned Border 2 Border motorized wheelchair ride this summer due to an unexpected surgery, but we are happy to report that he feels well enough to attempt a “Mini Border 2 Border” to scout out the route, which extends from the southern to northern borders of Washington state. He’d love to meet FSHers and invites you to make a donation to the FSH Society in support of his efforts. Learn more here.

Children’s Book Author Belinda Miller Supports the Society through Donations

Belinda Miller Ragwort ChroniclesBelinda Miller is a successful children’s book author who was diagnosed with facioscapulohumeral muscular dystrophy when she was 22 years old. Her diagnosis has not stopped her from doing the one thing she loves most: being an author. Belinda began her career as an elementary school language arts teacher, transitioning later to a career as an underwriter and legal assistant. She says that FSHD, being a slowly degenerating disease, allowed her to do a lot of living in a short amount of time. Continue reading

FSH Society Awards Grant to Establish Clinical Trial Research Network for FSHD

The FSH Society, a nonprofit, patient-driven organization that supports research and education for individuals with facioscapulohumeral muscular dystrophy (FSHD) and their families, today announced that it has awarded a $121,000 grant to co-principal investigators Jeffrey Statland, MD of the University of Kansas Medical Center, and Rabi Tawil, MD of the University of Rochester Medical Center, to expedite the development of new therapies for FSHD by developing a core FSHD Clinical Trial Research Network (CTRN). The FSH Society is funding four institutions that are piloting the CTRN: the University of Rochester, Kennedy Krieger Institute, Ohio State University, and the University of Kansas, all established centers with expertise in FSHD and neuromuscular clinical trials. Continue reading