January 18, 2019January 18, 2019
Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program
News
The U.S. Food and Drug Administration (FDA) selected Wave Life Sciences‘ Phase 2/3 trial assessing the efficacy and safety of WVE-210201 ... Read more
January 16, 2019January 16, 2019
Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD
News
Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular ... Read more
January 15, 2019January 14, 2019
I’ve Learned How to Deal with Judgment About My Disease
columns, You, Me and MD - a Column by Leah Leilani
In this day and age, when we have so much awareness about diversity, it surprises me how judgmental people ... Read more
January 14, 2019January 14, 2019
DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK
News
Until recently, the only two hospitals in the U.K. with the expertise to run Duchenne muscular dystrophy (DMD) ... Read more
January 11, 2019January 11, 2019
PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies
News
Kanneboyina Nagaraju, PhD, and his research team at Binghamton University, State University of New York will get a $329,000 ... Read more
January 9, 2019January 9, 2019
Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow
News
A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys with Duchenne muscular dystrophy ... Read more
January 7, 2019January 7, 2019
Capricor Places Hold on Phase 2 Trial of Cell Therapy After Patient Has Allergic Reaction
News
Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne muscular dystrophy (DMD) investigative cell therapy ... Read more
January 4, 2019January 4, 2019
Myonexus’ MYO-102 Earns FDA’s Orphan Drug Status for LGMD 2D
News
The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ MYO-102, a gene therapy for ... Read more
January 4, 2019January 4, 2019
Project Hercules Enlists Industry’s Help to Make Duchenne Therapies Affordable
News
Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business ... Read more
January 2, 2019January 2, 2019
Top 10 Muscular Dystrophy Articles of 2018
News
Throughout 2018, Muscular Dystrophy News Today provided daily coverage of new therapeutic strategies, clinical trials, and other topics ... Read more
December 21, 2018December 21, 2018
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
News
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same ... Read more
December 21, 2018December 21, 2018
PPMD Webinar Takes In-depth Look at MD Cardiac Disease and Recent Research Efforts
News
Magnetic Resonance Imaging (MRI) can catch signs of damage to the heart in muscular dystrophy (MD) patients earlier than ... Read more

January 18, 2019January 18, 2019

Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program

News

The U.S. Food and Drug Administration (FDA) selected Wave Life Sciences‘ Phase 2/3 trial assessing the efficacy and safety of WVE-210201 (suvodirsen) in Duchenne muscular dystrophy for its pilot program for complex innovative ... Read more

January 16, 2019January 16, 2019

Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD

News

Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Aldevron, which produces biological ... Read more

January 15, 2019January 14, 2019

I’ve Learned How to Deal with Judgment About My Disease

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

In this day and age, when we have so much awareness about diversity, it surprises me how judgmental people can still be. It was rare 60 years ago to ... Read more

January 14, 2019January 14, 2019

DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK

News

Until recently, the only two hospitals in the U.K. with the expertise to run Duchenne muscular dystrophy (DMD) clinical trials were turning studies away — they didn’t have the ... Read more

January 11, 2019January 11, 2019

PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies

News

Kanneboyina Nagaraju, PhD, and his research team at Binghamton University, State University of New York will get a $329,000 Parent Project Muscular Dystrophy (PPMD) grant to study responses to ... Read more

January 9, 2019January 9, 2019

Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow

News

A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys with Duchenne muscular dystrophy (DMD) at multiple sites in the U.S. and will soon open ... Read more

January 7, 2019January 7, 2019

Capricor Places Hold on Phase 2 Trial of Cell Therapy After Patient Has Allergic Reaction

News

Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne muscular dystrophy (DMD) investigative cell therapy CAP-1002 after a patient experienced a severe allergic reaction during blind ... Read more

January 4, 2019January 4, 2019

Myonexus’ MYO-102 Earns FDA’s Orphan Drug Status for LGMD 2D

News

The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ MYO-102, a gene therapy for limb-girdle muscular dystrophy type 2D (LGMD 2D), the company announced. The ... Read more

January 4, 2019January 4, 2019

Project Hercules Enlists Industry’s Help to Make Duchenne Therapies Affordable

News

Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the ... Read more

January 2, 2019January 2, 2019

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

December 21, 2018December 21, 2018

PPMD Webinar Takes In-depth Look at MD Cardiac Disease and Recent Research Efforts

News

Magnetic Resonance Imaging (MRI) can catch signs of damage to the heart in muscular dystrophy (MD) patients earlier than traditionally-used echocardiograms, according to a webinar providing updates on the ... Read more