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Rare Disease Day: Momentum in Neuromuscular Diseases is Building, but Progress Depends on Sustained Investment
Every year on Rare Disease Day, communities around the world come together to shine a
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A blind date with a book encourages me to dust off my social life
I’m going on a blind date! I should mention that it is with a book.
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Clinical Research Alert: Phase 2 Study of Rapcabtagene Autoleucel in Individuals with IIM
Researchers at Novartis are working to better understand idiopathic inflammatory myopathies (IIMs) and to evaluate a
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New research reveals protein pathway that can slow muscle repair
Researchers have identified a molecular pathway that helps regulate how muscle repairs itself — a
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25 Years Later: Reflecting on the 2001 MD-CARE Act Hearing and Where We Are Now
On Thursday, February 26, advocates, clinicians, patient leaders, and policymakers came together for a powerful
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Dreaming of solutions to the Olympic-sized challenges of FSHD
My family and I have never been athletically inclined. Our talents lie in other areas.
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Simply Stated: Updates in X-Linked Myotubular Myopathy (XLMTM)
X-linked myotubular myopathy (XLMTM) is a rare, inherited neuromuscular condition that primarily affects infant males.
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Roche halts development of satralizumab for DMD bone health
Roche has decided to stop developing satralizumab for bone health in Duchenne muscular dystrophy (DMD),
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Quest Podcast: Fashion for Every Body: Izzy Camilleri on Style, Function, and Inclusion
In this Quest Podcast episode, we chat with internationally recognized fashion designer Izzy Camilleri, a
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Progress Now: Research Updates and Breakthroughs
Amyotrophic lateral sclerosis (ALS) Phase 1 Clinical Trial: Recruiting This study, called LUMINA, is testing
