February 8, 2021February 4, 2021
It’s Time for Me to Go Outdoors and Take in the Vitamin D
Columns, Hawk's-Eye View - a Column by Hawken Miller
In the last year, it’s been easy for me to coop myself up in my room and spend the ... Read more
February 4, 2021February 4, 2021
Isometric Exercise in DMD Found to Improve Boys’ Strength, Function
News
Mild to moderate isometric leg exercise — contractions of a particular muscle or group of muscles — is safe ... Read more
February 3, 2021February 3, 2021
NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
News, syndicated
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a ... Read more
February 3, 2021February 1, 2021
Welcome to the Castle: Facing Down and Defeating Your Fears
Columns, Of Dragons and Dystrophin – a Column by Andy Rusch
His palms firmly pressed against the pommel, he holds the sword close to his chest. He can feel the ... Read more
February 2, 2021February 2, 2021
New App Plays ‘Pivotal’ Role in Increasing Registry Successes
News
In the first year since Parent Project Muscular Dystrophy (PPMD) and Thread launched a mobile app aimed at making ... Read more
January 28, 2021January 28, 2021
STRIVE Applications for Duchenne Community Now Open
News
Applications are now open for the seventh annual Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program for ... Read more
January 26, 2021January 26, 2021
PPMD Invests $1M in Satellos With Goal of Repairing Muscle Cells in Duchenne
News
Parent Project Muscular Dystrophy (PPMD) announced a $1 million investment in Satellos Bioscience to support the development of a ... Read more
January 25, 2021January 25, 2021
COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say
News, syndicated
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected ... Read more
January 25, 2021January 21, 2021
Why I’m Taking a Break From Playing Video Games
Columns, Hawk's-Eye View - a Column by Hawken Miller
I’m a self-described gamer, and proud of it, but lately I’ve realized I’ve probably been playing too much. Two ... Read more
January 21, 2021January 21, 2021
New CureDuchenne Initiative Provides Support, Education on COVID-19 Vaccination
News
CureDuchenne has launched a new initiative to prioritize the needs of the Duchenne muscular dystrophy (DMD) community during the COVID-19 ... Read more
January 20, 2021January 20, 2021
NORD’s Caregiver Respite Program Continues Through Pandemic
News, syndicated
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, ... Read more
January 19, 2021January 15, 2021
A Bittersweet Farewell to My Column Readers
Columns, You, Me and MD - a Column by Leah Leilani
Dear readers, This will be my last column for Muscular Dystrophy News. I want to thank you for ... Read more

December 10, 2020December 10, 2020

Translarna Approved in Russia for DMD Patients 2 and Older

News

Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients ages 2 and older. “Translarna was the first therapeutic ever approved ... Read more

December 8, 2020December 8, 2020

Lilly, Precision to Develop Gene Editing Therapies for DMD, Other Disorders

News

A new collaboration between Eli Lilly and Precision BioSciences aims to develop gene editing therapies for genetic disorders, initially focusing on Duchenne muscular dystrophy (DMD) and two other undisclosed ... Read more

December 8, 2020December 3, 2020

Give Yourself Some Peace of Mind This Holiday Season

Columns, You, Me and MD - a Column by Leah Leilani

This year has been full of seemingly insurmountable struggles for everyone. As we resolved one hardship, another seemed to suddenly appear, creating an endless cycle that rocked our mental ... Read more

December 7, 2020December 3, 2020

How to Avoid a Fall

Columns, Hawk's-Eye View - a Column by Hawken Miller

Having Duchenne muscular dystrophy is scary for a number of reasons. High up on that list is experiencing a fall. That’s especially true for those who have been on ... Read more

December 4, 2020December 4, 2020

MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

News, syndicated

The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention (CDC) to recommend that people living with neuromuscular diseases (NMDs) have early access to ... Read more

December 3, 2020December 3, 2020

ReveraGen Receives $3.3M NIH Grant to Advance Vamorolone in DMD

News

ReveraGen BioPharma has been awarded a $3.3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) to support further ... Read more

December 2, 2020December 2, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

December 1, 2020December 4, 2020

Sarepta Talks Progress, Plans for New Trials of DMD Gene Therapy SRP-9001

News

Note: This story was updated Dec. 4, 2020, to note that Teji Singh’s title is executive medical director of Sarepta, rather than the chief medical officer. Sarepta Therapeutics is ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 24, 2020November 20, 2020

Looking at Holiday Self-isolation in a Positive Light

Columns, You, Me and MD - a Column by Leah Leilani

The holidays are notorious for being one of the most hectic times of the year. People jump at the opportunity to score the best deals, and they sprint to ... Read more

November 24, 2020November 24, 2020

DM1 Patients Asked to Join Natural History Study Aiming for Better Clinical Trials

News

The Myotonic Dystrophy Clinical Research Network (DMCRN) is asking adults with myotonic dystrophy type 1 (DM1) to participate in a large natural history study aimed at improving clinical trials ... Read more

November 23, 2020November 19, 2020

Expressing Our Negative Emotions to Find Positive Ones

Columns, Hawk's-Eye View - a Column by Hawken Miller

I always hope to impart a positive message to my readers and exude joy in life whenever possible, but I also know it’s OK to be upset, even angry, ... Read more

It’s Time for Me to Go Outdoors and Take in the Vitamin D

Isometric Exercise in DMD Found to Improve Boys’ Strength, Function

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Welcome to the Castle: Facing Down and Defeating Your Fears

New App Plays ‘Pivotal’ Role in Increasing Registry Successes

STRIVE Applications for Duchenne Community Now Open

PPMD Invests $1M in Satellos With Goal of Repairing Muscle Cells in Duchenne

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Why I’m Taking a Break From Playing Video Games

New CureDuchenne Initiative Provides Support, Education on COVID-19 Vaccination

NORD’s Caregiver Respite Program Continues Through Pandemic

A Bittersweet Farewell to My Column Readers