The world's premier scientific meeting on facioscapulohumeral muscular dystrophy will highlight the latest on clinical trials and the need for more research on pediatric patients
RANDOLPH, Mass., June 15, 2023 /PRNewswire-PRWeb/ -- The FSHD Society's 30th annual International Research Congress (IRC) opened today in Milan Italy. The meeting gathers researchers, clinicians, patients and families, advocacy leaders, industry scientists, and regulators from around the world to focus on facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, hereditary muscle-weakening condition that affects an estimated one million men, women, and children worldwide. It is marked by life-long, progressive loss of muscle function and causes significant pain, fatigue, and disability. There is currently no medication to slow or halt the progression of symptoms.
Held this year on June 15-16, the IRC covers a broad span of research including the genetics, epigenetics, pathobiology, novel drug targets, outcome measures and clinical trials. The Society has funded research leading to the discovery of the genetic mechanism of FSHD, identification of therapeutic targets, and validation of clinical trial outcome measures. The Society also played a key role in launching the now 21-center international FSHD Clinical Trial Research Network, providing infrastructure for several ongoing clinical trials.
This year, the Society is holding a special session on pediatric FSHD. Most FSHD researchers and companies have focused on adult patients, even though the majority of people begin to manifest symptoms in childhood or adolescence. "We want to highlight research progress and gaps in our understanding of FSHD in children," says Jamshid Arjomand, PhD, chief science officer of the FSHD Society. "It's likely that future treatments will be most effective when given early in the course of the disease, so we need to be proactive to make sure the research is there to ensure that children will have access to treatments."
On the day preceding the IRC, the FSHD Society is hosting its annual Industry Collaborative Workshop for biopharmaceutical companies. The workshop will review the current state of trial readiness, including genetic testing, imaging markers, and clinical outcomes to seek alignment around approaches to addressing critical gaps.
The IRC will be followed on June 17-18 by the World FSHD Alliance's annual summit, a meeting of FSHD patient advocacy groups from 25 countries. With the prospect of new treatments coming on the market over the next few years, the alliance focuses on developing country-by-country strategies to ensure that patients everywhere will have access to these treatments.
"Our meetings serve a vital role in making sure that all stakeholders are working together to speed up the delivery of treatments to our families," said Arjomand.
About the FSHD Society
The FSHD Society is the world's largest research-focused patient organization for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy. The Society has catalyzed major advancements and is accelerating the development of treatments and a cure to end the pain, disability, and suffering endured by one million people worldwide who live with FSHD. The FSHD Society has transformed the landscape for FSHD research and is committed to making sure that no one faces this disease alone. The Society offers a community of support, news, and information through its website at https://www.fshsociety.org.
Media Contact
June Kinoshita, FSHD Society, 7813016060, [email protected]
SOURCE FSHD Society
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