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 potential new treatment for individuals living with IIM who have not responded to previous treatments (a condition known as refractory disease). The study Individuals with refractory IIM may be eligible to participate in a phase 2 clinical trial (AUTOGRAPH-IIM) evaluating…

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 light on conditions that are too often overlooked or underfunded. Historically, rare diseases have been defined by what they lack: large patient populations, widespread public awareness, and approved treatments. Yet collectively, rare diseases affect more than 30 million people in…

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 true pioneer in adaptive fashion. She shares how her successful career in high-end fashion took a transformative turn when she began designing clothing for people with disabilities and partnered with Silverts—work that helped ignite today’s adaptive fashion movement. Izzy shares…

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. It is one of the most severe forms within a group of disorders called centronuclear myopathies, which are characterized by distinctive muscle cell changes seen by biopsy and profound muscle weakness that begins early in life. It is estimated that…

What Is a VUS? Variants of Unknown Significance in Genetic Testing and Why They Matter

What should you do if you have a variant of unknown significance in genetic testing results? Hear from experts in the neuromuscular field.

Considering a Clinical Trial? 4 Things to Know Before You Enroll

Tips for participating in neuromuscular clinical trials and research studies, from MDA community members who have been there.

Expert Tips for Handling an Insurance Claim Denial for Gene Therapy

Follow these steps to appeal insurance denials for gene therapy, or any neuromuscular disease treatment, and improve your chances for approval.

Understanding Thymidine Kinase 2 Deficiency

Michio Hirano, MD, answers questions about TK2d, a rare mitochondrial myopathy with a new therapy approved by the FDA.

RNA Therapies Offer Keys to Treating Genetic Neuromuscular Diseases

RNA therapies offer new hope for neuromuscular diseases, targeting faulty proteins without permanently altering DNA.

Young Leader Living with Muscular Dystrophy Champions Aiming High and Setting Goals

Caroline LeMay, a Harvard graduate living with a disability, knows the importance of education to achieve her dreams.

Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. 

QUEST PODCAST

The Quest podcast, proudly presented by the Muscular Dystrophy Association, is part of the Quest family of content. Hosted by Quest Editor-in-Chief, motivational speaker and writer Mindy Henderson.

Episode 60- 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 true pioneer in adaptive fashion. She shares how her successful career in high-end fashion took a transformative turn when she began designing clothing for people with disabilities and partnered with Silverts—work that helped ignite today’s adaptive fashion movement. Izzy shares…

Episode 59 – Redesigning the Day: Accessibility and Mindset Life Hacks with Jax Cowles

In this Quest Podcast episode, we chat with public speaker, consultant, and disability advocate, Jax Cowles. Jax shares an honest, thoughtful, and deeply creative conversation about daily life, independence, and problem-solving.  She opens up about how creativity and “life hacking” became essential tools rather than optional skills, and how small, low-cost adaptations can completely transform…

Episode 58- Wrapping Up 2025 with Ira and Lily

In this Quest Podcast episode, we chat with Muscular Dystrophy Association’s National Ambassadors, Lily S. and Ira Walker. Lily is a dedicated advocate finishing her first year as a National Ambassador. She  shares her journey, why she believes it is important to advocate for yourself and others, and what she has learned along the way….