6 articles from the last 30 days matching "Duchenne or Becker Muscular Dystrophy"
PipelineRSSToday
Two companies, Epicrispr Biotechnologies and Forge Biologics, are working together to make EPI-321, a new one-time treatment for facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare muscle disease that causes progressive weakness. The treatment works by using epigenetics—a way to turn off genes without changing their DNA—to stop the abnormal gene that causes FSHD.
WHY IT MATTERSThis partnership could accelerate production of EPI-321, potentially bringing a one-time treatment option closer to FSHD patients who currently have limited approved therapies.
AdvocacyRSSApr 30
CureDuchenne is hosting a four-day conference in Orlando from May 21-24 where families and patients with Duchenne and Becker muscular dystrophy can learn about the latest research, new treatments, and connect with others in the community. The event aims to provide education, support, and hope to people affected by these muscle-weakening conditions.
WHY IT MATTERSThis conference offers patients and families direct access to the latest research updates and treatment options for Duchenne and Becker muscular dystrophy, plus the chance to connect with others managing these progressive conditions.
Clinical trialCLINICALTRIALSApr 30
Researchers are studying a new care model designed to help patients with complex rare diseases get better coordinated treatment and support. The trial involves 136 patients with conditions like mitochondrial diseases and muscular dystrophies. The goal is to bridge the gap between scientific discoveries and actual patient care by creating a more organized system for managing these complicated conditions.
WHY IT MATTERSThis trial is testing a coordinated care approach specifically for patients with multiple rare diseases or complex symptoms, which could improve how you receive care across different specialists and reduce the fragmentation many rare disease patients experience.
AdvocacyRSSApr 29
A person with facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle disease, is sharing their experience of losing mobility and needing a wheelchair. FSHD gradually weakens muscles over time, but it's hard to predict how fast the disease will progress in each person. This person's situation was made more complicated by injuries from a car accident years ago that damaged their joints.
WHY IT MATTERSFSHD patients face unpredictable disease progression that can rapidly impact mobility and independence, making adaptive equipment planning and financial preparation for accessibility needs critical but difficult.
NewsRSSApr 22
A person with facioscapulohumeral muscular dystrophy (FSHD) is finding it harder to move around their home with a walker and is exploring other mobility options. They recently visited different places to look for solutions that might work better for their situation. This article discusses the process of finding new mobility devices when current ones stop working well enough.
WHY IT MATTERSPeople with FSHD often face progressive mobility challenges, and knowing what alternative devices exist can help maintain independence and quality of life as the disease progresses.
AdvocacyRSSApr 22
Three organizations that work with facioscapulohumeral muscular dystrophy (FSHD) patients are joining together to make clinical trials better. FSHD is a rare muscle disease that causes weakness in the face, shoulders, and upper arms. This partnership wants to improve how these trials are designed so they can test new treatments more effectively.
WHY IT MATTERSBetter-designed clinical trials mean faster progress toward treatments for FSHD, and patient input through the FSHD Society ensures trials are structured in ways that actually work for people living with the disease.