DMD Features

DMD Features, DS Features, Features

Tevard Biosciences Pursues Gene Therapy to Treat Dravet Syndrome and Possibly DMD

By

Larry Luxner

Publish Date August 22, 2023

Tevard Biosciences aims to use transfer RNA (tRNA) to modulate RNA function to treat Dravet syndrome and other rare diseases.

DMD Features, Features

Texas Family Raises Millions for Groundbreaking Duchenne Research

By

Larry Luxner

Publish Date August 21, 2023

Tim and Laura Revell have raised millions of dollars for CureDuchenne to research a disease that affects both their sons.

CF Features, DMD Features, Features, HAE Features, HD Features, HDFN Features, PWS Features, SMA Features

Experts Urge Rare Disease Screening for Everyone, Not Just Pregnant Women

By

Larry Luxner

Publish Date July 31, 2023

The American College of Obstetricians and Gynecologists now recommends that all women—ideally before pregnancy—be screened for hemoglobinopathies.

DMD Features, Features

Edgewise and Locanabio Target New Mechanisms for Treating DMD

By

Larry Luxner

Publish Date July 31, 2023

Edgewise Therapeutics focuses on small-molecule therapies to preserve muscle in patients with DMD, while Locanabio is working on snRNA technology.

Conferences, DMD Features, Features, MDA 2023, SMA Features

SMA Patient and Advocate Mindy Henderson Educates Others as Editor of MDA Magazine

By

Larry Luxner

Publish Date June 9, 2023

Mindy Henderson, the new editor-in-chief of the Muscular Dystrophy Association’s Quest magazine, has a lifelong association with the group.

DMD Features, Features

Pfizer Gene Therapy Shows Huge Promise for Boys With DMD, But Questions Loom

By

Larry Luxner

Publish Date May 25, 2023

Two boys with DMD—one in Connecticut and one in California—have shown remarkable results in the trial of a gene therapy from Pfizer.

DMD Features, Features

Texas Researcher Exploring CBD as a Potential Therapy for Duchenne  

By

Larry Luxner

Publish Date May 19, 2023

A researcher at Baylor College of Medicine is studying whether cannabidiol (CBD) could help boys and young men who have DMD.

DMD Features, Features

Neuromuscular Clinics Expect Surge in Demand if FDA Approves DMD Gene Therapy

By

Larry Luxner

Publish Date May 4, 2023

Julie Parsons, MD, of Children’s Hospital Colorado, expects a surge in demand if the Food and Drug Administration approves a new gene therapy for Duchenne.

DMD Features, Features

Little Hercules Foundation’s Newest Mission: DMD Screening in All 50 States

By

Larry Luxner

Publish Date April 11, 2023

Randi Clites of Little Hercules Foundation has made it her mission to get DMD newborn screening implemented in all 50 states.

DMD Features, Features

Nation’s First CureDuchenne Clinic Opens in Texas for Hispanic Patients With DMD

By

Larry Luxner

Publish Date March 28, 2023

The nation’s first CureDuchenne Clinic has opened in a suburb of Dallas, Texas, to treat Hispanic patients with rare neuromuscular diseases.