FSHD-Global Research Foundation

11/06/2026

Encouraging news from the Avidity/Novartis trial using targeted siRNA to suppress DUX4 in . The therapy, del-brax, is an investigational antibody oligonucleotide conjugate (AOC) – a new class of RNA therapeutics – showing potential to become the first disease-modifying treatment for FSHD.

Del-brax is designed to address the root cause of FSHD, the aberrant expression of DUX4, in previously hard-to-reach muscle cells.

The FORTITUDE Phase I/II study (NCT05747924) is a randomised, double-blind, placebo-controlled clinical trial evaluating safety, dosage, and exploring efficacy. The FORTITUDE study met its endpoints: a change in plasma concentration of KHDC1L, a DUX4-regulated circulating biomarker, and a change from baseline in the levels of creatine kinase, a marker of muscle damage.

This is an encouraging and vital step in the research, but it still needs to be shown that del-brax has an impact on disease in FSHD patients, beyond those two biomarkers. Phase III clinical trials are now underway in the US and Europe to evaluate the effects further.

We will keep you updated. Join our Cure FSHD Registry to ensure you are trial ready to take advantage of future progress in this or other FSHD therapy trials in Australia.

Join the Cure FSHD Patient Registry here: https://fshd-app.weguide.com.au/

📙Read the Novartis announcement here: https://www.novartis.com/news/media-releases/novartis-delpacibart-braxlosiran-del-brax-phase-iii-study-facioscapulohumeral-muscular-dystrophy-fshd-meets-primary-biomarker-endpoint?fbclid=IwY2xjawSXVW5leHRuA2FlbQIxMABicmlkETFEYm5aM2JuTUREeGpuNE9zc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHsosx0vnBqHH5Kp7cKKY8q86_BD3kE4SeUYdDOQr2ro9Ww_Ab-I49g2-60TL_aem_q-51PGRw2GO4s3I1QLkIKQ

03/06/2026

World FSHD Day is coming June 20! What will you do to raise awareness for FSH muscular dystrophy? in Coffs Harbour will be lighting up orange in support! Reach out to your local council and landmarks to get them to do the same. You'll find a letter template, social tiles and more in the World FSHD Day toolkit. Visit: https://fshdglobal.org/news-events/world-fshd-day/

03/06/2026

Our Project Mercury poster is up at the ANN-AOMC Joint Congress in Perth! We are very excited to share more about this patient-led, global initiative that operates across ten countries to speed up clinical trials and development of therapies for FSHD. If you haven't yet purchased a ticket for the patient community day of the conference on Friday, there's still time (use 75% off discount code: NMWA): https://eecw.eventsair.com/ann-aomc-joint-congress-2026

01/06/2026

Congratulations to Peter and Takako Jones Lab on their publication describing the saliva testing they developed for FSHD. Every Australian who has gone through our home-based testing is represented in this research. Thank you for helping make this possible!

Whether you participated in testing or donated funds to help progress the research and make the testing more affordable - you should all be proud of this outcome. This publication will enable more research and will help raise awareness in the scientific and medical communities.

At home saliva-testing for FSHD is no-cost to our Cure FSHD patient registry participants, so please join the registry if you believe you or a family member may be affected. Join 'Cure FSHD' using the button on our website: https://fshdglobal.org/

Link to publication: https://www.biorxiv.org/content/10.64898/2026.05.26.727947v1?fbclid=IwZnRzaASIOJpleHRuA2FlbQIxMQBzcnRjBmFwcF9pZAo2NjI4NTY4Mzc5AAEe6TQkXWw-A_cjPBAaRsnBs4BZTQS5B6zAq89nU4HIrlBYoND-SifdSAlX7o0_aem_YfO0HNARFkclc8fnfPdZkg

30/05/2026

Just 6 DAYS AWAY! The ANN-AOMC Joint Congress in Perth on the 5th of June is fast approaching. Don't forget to register for the community day. Details below...

Conference Page: https://eecw.eventsair.com/ann-aomc-joint-congress-2026/

When you register, use 75% off discount code: NMWA
NOTE: Select registration for the Community/Patient Day Only (this ticket does not include the 3rd and 4th June)

There is a limit to the allocation of partially funded tickets, and they will be fulfilled on a first-in first-served basis, so to avoid disappointment, please don't delay registering.

To see all that is on offer for you to be a part of, please read the attached program for the Community/Patient Day.

29/05/2026

Thank you to our guests, speakers, and sponsors for a magnificent event in Melbourne today! We have come so far in support of those with FSHD muscular dystrophy and will be able to do so much more thanks to your contributions.

29/05/2026

To everyone who missed this fantastic and webinar, featuring the latest on FSHD research and clinical trials, the recording is now available to watch here: https://fshdglobal.org/neuromuscular-disease-webinar/

We’re pleased to be joining Muscular Dystrophy Queensland for an upcoming webinar on Tuesday, 19 May.

Our CEO and Managing Director, Emma Weatherley, will present on the latest developments in FSHD research and the Clinical Trial Passport, including what’s on the horizon for the community. She will be joining a range of speakers from across the neuromuscular sector.

We look forward to being part of this important conversation and connecting with the neuromuscular community.

📅 Tuesday 19 May
🕙 10.00am - 2.30pm (AEST)
💻 Online via Microsoft Teams

🔗 For topics, speakers and to register, click here: https://mdqld.org.au/events/mdq-neuromuscular-seminar/

27/05/2026

Research highlighted today in the journal Neuromuscular Disorders describes some of the incredible research made possible by FSHD Global Research Foundation, thanks to our supporters, and especially thanks to our Cure FSHD patient registry participants. Your voices inform research that is fast-tracking the development of therapies and helping to bring clinical trials to Australia. Read more about this research here:

A review of the pioneering research that uses MRI scans and AI to enable clinical trials for FSH muscular dystrophy and other neuromuscular conditions.

26/05/2026

Tickets close at midnight tonight! This is your last chance Melbourne to enjoy a Luxe Lunch hosted by FSHD Global Research Foundation. Enjoy fine dining, champagne, and exclusive silent auction opportunities – including a 7-night escape or a private executive gym membership and more – while networking with innovative founders and business leaders in an elegant setting. You’ll also be supporting life-changing research into FSHD muscular dystrophy. Get your tickets here: https://fshdglobal.org/luxe-lunch/

22/05/2026

A warm welcome to Valles Accountants, the newest sponsor for our Luxe Lunch in Melbourne on Friday, 29 May. Their generous support helps fund critical research and drives progress towards treatments and a cure for FSHD. There is still time to join them as a sponsor or to purchase tickets to what promises to be a fabulous event. https://fshdglobal.org/luxe-lunch/