Learn more about our positive long-term MARINA-OLE™ del-desiran (AOC 1001) data including vHOT video and patient testimonials

Russell’s Story

At age 15, Russell received a long-awaited diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD). After two years of seeing various specialists, wondering what could explain his symptoms, and constant invalidation from healthcare providers, Russell finally received an explanation for his pain and limitations. In some ways, Russell found relief in his diagnosis as he could finally explain his condition to others and seek appropriate accommodations in school and athletics. Yet, at the same time, facing a rare and progressive muscle disease at a young age was daunting and heartbreaking.

The hardest part of my diagnosis was that I had dysfunction, but I wasn’t honest with myself that I had limitations. And because I wasn’t honest with myself, I couldn’t be honest with others.

While coping with an FSHD diagnosis was challenging, Russell chose to channel his frustration into another avenue: advocacy. Russell dove in and decided to become an integral part of the FSHD community. As a high school volleyball player, Russell and his team founded an annual event to fundraise for FSHD research and raise awareness. Shortly after, Russell became connected with the FSHD Society, the world’s largest research-focused patient organization for FSHD. Russell is now an ambassador for the FSHD Society and connects with newly diagnosed people to share his journey with others his age.

When Russell reached college, he found new avenues to further his commitment as he gained experience researching muscular dystrophies in a laboratory, including FSHD and Duchenne muscular dystrophy. Motivated by a long and frustrating diagnostic journey, Russell is attending medical school with the hopes of using his platform as a future physician to elevate the voices of people living with rare diseases and to ensure that their needs are met.

One of the core reasons I wanted to go to medical school to become a doctor was to prevent others from experiencing the challenging diagnostic odyssey that I endured.

While Russell strives to maintain a positive outlook, he faces challenges with pain and everyday tasks. As a result, asking for accommodations has become a persistent component of his professional and personal life. In addition, explaining his condition can be emotionally exhausting as people often discredit his diagnosis because not all of his limitations are visible to others.

In a lot of ways, FSHD can be an invisible diagnosis. A lot of people don’t know from looking at me that I have a muscular dystrophy or that I have difficulties with certain tasks and aspects of daily living.

Despite these everyday challenges, Russell remains determined not to let FSHD weigh him down. Today, he enjoys hiking, fishing, reading, and traveling in his spare time. He is hopeful that soon there will be a treatment and ultimately, a cure for the condition. A few key ways Russell maintains a balance and manages his symptoms involve listening to his body and being conscious of certain parts of his body becoming overworked. Meeting with a physical therapist has been essential to achieving this balance while ensuring his actions are smart and safe. Sharing his practices and what has worked for him remains paramount as he connects with new patients and encourages them to keep moving forward.

Be honest with yourself and don’t give up hope. There’s research happening and there are things you can do to try to help yourself. You have to be your own advocate.

Arthur A. Levin, PhD

Arthur Levin, a founding member of Avidity, serves as Distinguished Scientist and Strategic Leader at Avidity Biosciences and is a member of the Board of Directors. He previously held the position of Chief Scientific Officer at Avidity. Dr. Levin is a key opinion leader in the RNA therapeutics field who has led teams responsible for the development of many oligonucleotides. Prior to joining Avidity, Dr. Levin was the Executive Vice President of Research and Development at miRagen Therapeutics and held senior drug development roles at Ionis (formerly Isis) Pharmaceuticals and Santaris Pharma.

Dr. Levin has played key roles in the development of numerous oligonucleotides including the first approved antisense drugs and the first microRNA-targeted therapeutic in clinical trials. He has a combined three decades of experience in all aspects of drug development from discovery through drug registration, both in large pharma and biotech companies. Dr. Levin has published more than 100 scientific articles and several of the most cited reviews in the field. He is on the scientific advisory boards of multiple institutions. Art received a doctorate in toxicology from the University of Rochester, and a bachelor’s degree in biology from Muhlenberg College.

2022 World Muscle Society Congress

A Phase 1/2 Clinical Trial Evaluating the Safety and Pharmacokinetics of AOC 1001 in Adults with Myotonic Dystrophy Type 1: MARINA Study Design

AOC 1020: An Antibody Oligonucleotide Conjugate (AOC) in Development for the Treatment of FSHD

Durable AOC Mediated Exon 44 Skipping In Non-Human Primate Muscle Tissue And Dystrophin Protein Restoration In DMD Patient Derived Skeletal Muscle Cells

Prevalence of Healthcare Conditions and Services Used by Patients with Myotonic Dystrophy (DM) Pre- and Post-Diagnosis: A Real-World Data Analysis

Understanding the Patients’ Journey Pre- and Post-Diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD): A Real-World Retrospective Data Analysis

2022 74th AAN Annual Meeting

Study Design of AOC 1001-CS1, a Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA™) 

Antibody-Oligonucleotide Conjugates (AOCs) Demonstrate Potent and Durable Exon Skipping and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy

Prevalence of healthcare conditions and services used by patients with myotonic dystrophy (DM1 and DM2) prior to diagnosis; a real-world data analysis

Understanding the patients’ journey to the diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD): a real-world retrospective data analysis

DUX4 siRNA Optimization for the Development of an Antibody-Oligonucleotide Conjugate (AOC™) for the Treatment of FSHD

2022 MDA Clinical and Scientific Conference

DUX4 siRNA Optimization for the Development of an Antibody-Oligonucleotide Conjugate (AOCTM) for the Treatment of FSHD

A Phase 1/2 Clinical Trial Evaluating the Safety and Pharmacokinetics of AOC 1001 in Adults with Myotonic Dystrophy Type 1 (DM1): MARINA Study Design

Understanding the patients’ journey pre- and post-diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD): a real-world retrospective data analysis 

Prevalence of healthcare conditions and services used by patients with myotonic dystrophy pre-and post-diagnosis, a real-world data analysis 

Kathleen Gallagher

Kathleen Gallagher joined Avidity Biosciences in April 2021 and serves as Senior Vice President and Global Program Head, Myotonic Dystrophy Type 1 (DM1). She previously held the role of Senior Vice President of Corporate Communications and Investor Relations where she built the foundational communications strategy for Avidity. Ms. Gallagher brings more than 20 years of experience in the biopharmaceutical industry leading communications and investor relations in preclinical to commercial stage companies. Prior to joining Avidity, she was Vice President, Investor Relations and Corporate Communications at Akcea Therapeutics (acquired by Ionis Pharmaceuticals), where her communications strategy and relationship building expertise were essential through the development and commercial launches of rare disease therapies Tegsedi® and Waylivra®. Previously, she held roles of increasing responsibility during her 13-year tenure at Merrimack Pharmaceuticals, where she provided leadership and direction through multiple financing rounds including an IPO and through the launch of the pancreatic cancer therapy ONIVYDE®. Ms. Gallagher earned her B.S. degree in English from Boston University.