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 ... 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 ... 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 ... 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 ... Read more
November 19, 2020November 19, 2020
Phase 1 Trial of Oral EDG-5506 for Becker MD Enrolling at Texas Site
News
Edgewise Therapeutics announced the opening of a Phase 1 clinical trial to assess the safety and tolerability of EDG-5506, ... Read more
November 18, 2020November 18, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
News, syndicated
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how ... Read more
November 17, 2020November 17, 2020
Q&A: Meet the Muscular Dystrophy Association’s New CEO and President
News
The Muscular Dystrophy Association (MDA) announced last week it had selected former MDA vice chairman of the board Donald ... Read more
November 12, 2020November 12, 2020
MDA Launches Tool to Make Clinical Data More Accessible to Researchers
News
The Muscular Dystrophy Association (MDA) and DNAnexus have introduced a digital platform to improve patient care and accelerate treatment discovery ... Read more
November 10, 2020November 6, 2020
Saying Goodbye to Eye Strain, Thanks to Blue-light Glasses
columns, You, Me and MD - a Column by Leah Leilani
Technology is an essential tool I use both in my social life and to be more productive at work. ... Read more
November 10, 2020November 10, 2020
AMO-02 for Congenital DM1, Entering Pivotal Trial, Gets Rare Pediatric Disease Status
News
The U.S. Food and Drug Administration (FDA) has given rare pediatric disease status to AMO Pharma‘s investigational therapy AMO-02 (tideglusib), now entering ... Read more
November 9, 2020November 5, 2020
How I’m Getting Out of My 2020 Funk
columns, Hawk's-Eye View - a Column by Hawken Miller
If 2020 hasn’t been a challenging year for you, that’s great news. But for me, like many others, this ... Read more
November 5, 2020November 5, 2020
Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy
News
Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne muscular ... 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

November 19, 2020November 19, 2020

Phase 1 Trial of Oral EDG-5506 for Becker MD Enrolling at Texas Site

News

Edgewise Therapeutics announced the opening of a Phase 1 clinical trial to assess the safety and tolerability of EDG-5506, its investigational oral treatment for Becker muscular dystrophy (BMD). The ... Read more

November 18, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

November 17, 2020November 17, 2020

Q&A: Meet the Muscular Dystrophy Association’s New CEO and President

News

The Muscular Dystrophy Association (MDA) announced last week it had selected former MDA vice chairman of the board Donald Wood, PhD, as the new president and CEO of the ... Read more

November 12, 2020November 12, 2020

MDA Launches Tool to Make Clinical Data More Accessible to Researchers

News

The Muscular Dystrophy Association (MDA) and DNAnexus have introduced a digital platform to improve patient care and accelerate treatment discovery for muscular dystrophy and other neuromuscular disorders. The neuroMuscular ObserVational ... Read more

November 10, 2020November 6, 2020

Saying Goodbye to Eye Strain, Thanks to Blue-light Glasses

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

Technology is an essential tool I use both in my social life and to be more productive at work. While it can make certain things I struggle with easier, ... Read more

November 10, 2020November 10, 2020

AMO-02 for Congenital DM1, Entering Pivotal Trial, Gets Rare Pediatric Disease Status

News

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease status to AMO Pharma‘s investigational therapy AMO-02 (tideglusib), now entering clinical testing in children and adolescents with congenital myotonic dystrophy ... Read more

November 9, 2020November 5, 2020

How I’m Getting Out of My 2020 Funk

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

If 2020 hasn’t been a challenging year for you, that’s great news. But for me, like many others, this year has been a lesson in overcoming adversity, whether it’s ... Read more

November 5, 2020November 5, 2020

Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy

News

Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne muscular dystrophy (DMD). The collaboration will combine Solid’s gene therapy construct for ... Read more