-
PTC Therapeutics Shares Updated Community Letter Regarding Ataluren
Today PTC Therapeutics released an updated letter to the Duchenne community on their website with
-
Napa in Newport Raises $1.6 Million to Accelerate Research for Duchenne Muscular Dystrophy
Renowned Napa Valley Vintners and Philanthropists Unite in Newport Beach for a Weekend of Wine, Food,
-
Bridgebio asks FDA to approve potential 1st treatment for LGMD2i
Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to
-
Simply Stated: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare neurological disorder in which the immune system
-
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
