DMD
What Is Duchenne Muscular Dystrophy (DMD)?: 108 FAQs
Duchenne muscular dystrophy (DMD), commonly known as Duchenne, is a rare genetic disorder that causes progressive muscle weakness and degeneration. It is caused by mutations in the DMD gene, which affect the body's ability to produce functional dystrophin, a protein essential for protecting muscle fibers. Symptoms often appear in...
DMD
What Is Exon Deletion? Understanding Exon Deletions in Duchenne Muscular Dystrophy
What is exon deletion, and why does it matter in Duchenne muscular dystrophy? An exon deletion occurs when one or more sections of a gene are missing, potentially affecting how the body produces an essential protein. In DMD, these deletions occur in the DMD gene, which contains the instructions...
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DMD
What Are mdx Mice, and Why Are They Used in Research of Duchenne Muscular Dystrophy (DMD)?
The mdx mouse is the most widely used animal model for studying Duchenne Muscular...
DMD
What Is Prenatal Testing for Duchenne Muscular Dystrophy (DMD)?
Prenatal testing for Duchenne Muscular Dystrophy (DMD) helps expectant parents understand whether their unborn...
DMD
Biologics License Application (BLA): A Complete Guide to the FDA Approval Process
The Biologics License Application (BLA) plays a critical role in bringing new biologic therapies...
DMD
May Becker Patients Receive Elevidys? Does Elevidys Benefit Becker Patients?
Is Becker muscular dystrophy eligible for Elevidys? In this article, we’ll explore current research,...
DMD
North Star Ambulatory Assessment (NSAA) in Duchenne Muscular Dystrophy (DMD)
The North Star Ambulatory Assessment (NSAA) is a standardized, clinician-administered tool designed to evaluate...
DMD
What is Creatine Kinase (CK)? What Does High Creatine Kinase Mean?
Creatine Kinase (CK), also known as creatine phosphokinase (CPK), is an enzyme found in...
DMD
Mutations and Deletions Amenable to Exon 53 Skipping Therapies for Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a debilitating and progressive genetic disorder that primarily affects...
DMD
Mutations and Deletions Amenable to Exon 51 Skipping Therapies for Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder caused by mutations in the...
DMD
Micro Dystrophin: 5 Critical Questions Everyone Is Thinking About
Micro dystrophin and full-length dystrophin play crucial roles in treating Duchenne muscular dystrophy (DMD),...
DMD
What is Cytomegalovirus Infection (CMV)?
Sarepta shares that a teenager with Duchenne muscular dystrophy has passed away following treatment...
DMD
What does DMD Warrior mean?
A DMD Warrior (aka Duchenne Warrior) is a term used to describe individuals who...
Latest articles
DMD
What Is Duchenne Muscular Dystrophy (DMD)?: 108 FAQs
Duchenne muscular dystrophy (DMD), commonly known as Duchenne, is a rare genetic disorder that...
Press Releases
Entrada Therapeutics ENTR-601-45: Early Exon 45 Skipping Study Shows Increased Dystrophin Production
Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201,...
Press Releases
Entrada Therapeutics Advances ENTR-601-44 to 18 mg/kg in Its Duchenne Exon 44 Skipping Study
Entrada Therapeutics has received clearance to test the highest dose in its Exon 44...
Research
Sapreme Therapeutics Reports Strong Preclinical Dystrophin Restoration With SPM-2551 in Duchenne
Sapreme Therapeutics has announced promising preclinical results for SPM-2551, an experimental treatment for Duchenne...
Press Releases
Deramiocel Shows Positive 24-Month HOPE-3 Results in Duchenne
Capricor Therapeutics has reported new results from the HOPE-3 study evaluating Deramiocel in people...
Press Releases
Ractigen Therapeutics Reports Positive First-in-Human Data for RAG-18 in Duchenne
Ractigen Therapeutics has presented positive first-in-human data for RAG-18 (NCT07282652), an investigational treatment for...
Press Releases
Entrada Therapeutics’ ENTR-601-44 Shows Dose-Dependent Exon 44 Skipping in Early PK/PD Data
Entrada Therapeutics’ ENTR-601-44 is an investigational therapy designed for patients with Duchenne muscular dystrophy...
Press Releases
NS Pharma’s Brogidirsen Shows 5-Year Clinical Data in DMD Patients Amenable to Exon 44 Skipping
NS Pharma’s brogidirsen (NS-089/NCNP-02) is an investigational therapy for Duchenne muscular dystrophy (DMD) designed...