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Monica Torres is a 42-year-old mom of 6. Monica was born and raised in Puerto Rico and recently moved to the city of New York. She has a service dog…

Researchers at participating clinical research sites are seeking individuals with amyotrophic lateral sclerosis (ALS) for a phase 3 study (PREVAiLS). This study will evaluate the safety and efficacy of the…

Q&A with Fiona Cauley about her comedy, accessibility challenges, and advocacy living with Friedreich ataxia (FA).

Carlee Weber and Nicole Lucas This month, the Muscular Dystrophy Association (MDA) is launching an important advocacy campaign urging Congress to advance policies that improve the lives of family caregivers…

CIAO1-related neuromuscular disorder is an inherited condition that was first reported on in late 2023. It is caused by variants in the CIAO1 gene and primarily affects muscle function, though…

Julianne Meiser, MSS, LCSW, an Outpatient Social Worker in the Division of Neurology at the Children’s Hospital of Philadelphia. By nature, medical emergencies are unexpected, stressful, and often traumatic experiences.…

Researchers at Biogen are seeking pediatric FA patients to participate in a phase 3 clinical trial to better understand Friedreich ataxia (FA) and to evaluate omaveloxolone, an investigational therapy for…

What happens after a drug is approved? Sometimes research continues to make it more effective, convenient, or targeted to a neuromuscular disease.

Bio: Rebecca Hume is a Senior Specialist Writer for Quest Media. She graduated from Slippery Rock University and worked in the Pennsylvania Medicaid Waiver Program for more than 10 years…

Researchers at Shionogi Inc. are conducting a phase 2 clinical trial for adults with late-onset Pompe disease (LOPD) who are receiving standard-of-care enzyme replacement therapy (ERT). The study will evaluate…