-
Letter from the Editor: How New Higher-Dose Regimen for Spinraza Brings More Hope
Mindy Henderson, MDA’s Vice President of Disability Outreach & Empowerment & Editor-in-Chief of Quest Media
-
Life with Lily: Why an Expert Specialist is Crucial for Your Care
Living with a rare disease means navigating a healthcare system that often feels fragmented and
-
I’m grateful for meaningful conversations with my adult children
As parents, we dedicate our lives to raising our children. We work long and hard,
-
Early trial data support RNA therapies for two muscular dystrophy types
SRP-1001 and SRP-1003, Sarepta Therapeutics’ investigational RNA-based therapies for two types of muscular dystrophy, were
-
Quest Podcast: Pizazz, Purpose, and Periodic Paralysis: How Cienna Ditri Turns Lived Experience into Advocacy
In this Quest Podcast episode, we chat with advocate, social media influencer, and President of
-
Figuring out where to buy a lift chair for my FSHD symptoms was no small task
Recently, standing up has become even more difficult for me than it previously was. The
-
Wildland Fire Fighter Refuses to Allow Diagnosis to Define His Life
When Tyler Long was diagnosed with facioscapulohumeral muscular dystrophy (FSHD) during his senior year of
-
Sarepta plans to seek full FDA approval of 2 Duchenne treatments
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full
-
PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy
PPMD and Secretome Therapeutics (Secretome) are excited to announce that PPMD has provided $250,000 in
-
When it comes to rare disease advocacy, just keep showing up
According to the EveryLife Foundation for Rare Diseases, more than 30 million Americans live with
