Updates

February 15, 2019February 15, 2019
Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA

Sarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen (SRP-4053), a potential treatment for ... Read more
February 13, 2019February 13, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
February 13, 2019February 13, 2019
Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019

With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy (DMD) diagnosis, Parent Project Muscular Dystrophy (PPMD) continues its multi-year ... Read more
February 12, 2019February 8, 2019
Tips to Alleviate Symptoms of Constipation

Managing one’s health while living with a disability requires much more than regular trips to the doctor and excessive amounts of medications. For those ... Read more
February 11, 2019February 11, 2019
Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces

Capricor Therapeutics announced that patient dosing has resumed in its HOPE-2 trial of CAP-1002, an investigative cell therapy for Duchenne muscular dystrophy The company placed ... Read more

News

Defying the Odds of Living with Duchenne, Decade After Decade
February 8, 2019February 14, 2019
When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the ...
News

PPMD Funds Gene Therapy Research Aimed at Improving Cardiac Function in DMD Patients
February 6, 2019February 14, 2019
Parent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School ...
columns, You, Me and MD - a Column by Leah Leilani

I Can’t Do Everything, and That’s OK
February 5, 2019February 12, 2019
A couple weeks ago, a blogger with mitochondrial myopathy posted an interesting piece of advice ...
News

ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
February 5, 2019February 6, 2019
G71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, ...
News

DMD Increases Susceptibility to Rare, Aggressive Muscle Cancer, Mouse Study Suggests
February 4, 2019February 7, 2019
Severe Duchenne muscular dystrophy (DMD) is linked to increased susceptibility to a rare and ...
News

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
February 1, 2019February 6, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...

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Popeyes Group Launches 15th Annual Campaign to Help MDA
February 1, 2019February 7, 2019
Popeyes Louisiana Kitchen’s franchisee Sailormen has launched the 15th Annual “Appetite for a Cure” campaign ...
ICER Document Reviews Effectiveness of 3 Duchenne MD Therapies
January 30, 2019February 6, 2019
The Institute for Clinical and Economic Review (ICER) has set up a draft document to ...
Finding the Perfect Wheelchair Blanket
January 29, 2019January 28, 2019
Phase 2b Trial Testing Vamorolone Now Enrolling Boys Ages 4-6 with DMD
January 28, 2019February 12, 2019
Connecticut Man With Limb-girdle MD Inspires Others
January 25, 2019February 13, 2019
PPMD Publishes 10-Year Duchenne and Becker Patient Registry Report
January 23, 2019January 23, 2019

Seeing Both Sides of ‘The Upside’
January 22, 2019January 28, 2019
Spoiler alert: This column reveals some plot details of the newly released film “The Upside.” ...
New Tool to Help With Virtual Research and Care Management for Duchenne Patients
January 21, 2019January 21, 2019
Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program
January 18, 2019January 18, 2019
Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD
January 16, 2019January 16, 2019

I’ve Learned How to Deal with Judgment About My Disease
January 15, 2019January 14, 2019
In this day and age, when we have so much awareness about diversity, it ...
DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK
January 14, 2019January 14, 2019
PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies
January 11, 2019January 11, 2019

News

Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow
January 9, 2019January 9, 2019
A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys ...
News

Capricor Places Hold on Phase 2 Trial of Cell Therapy After Patient Has Allergic Reaction
January 7, 2019January 7, 2019
Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne ...
News

Myonexus’ MYO-102 Earns FDA’s Orphan Drug Status for LGMD 2D
January 4, 2019January 4, 2019
The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ ...
News

Project Hercules Enlists Industry’s Help to Make Duchenne Therapies Affordable
January 4, 2019January 4, 2019
Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter ...
News

Top 10 Muscular Dystrophy Articles of 2018
January 2, 2019January 2, 2019
Throughout 2018, Muscular Dystrophy News Today provided daily coverage of new therapeutic strategies, clinical trials, ...
News

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 21, 2018December 21, 2018
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures ...
News

PPMD Webinar Takes In-depth Look at MD Cardiac Disease and Recent Research Efforts
December 21, 2018December 21, 2018
Magnetic Resonance Imaging (MRI) can catch signs of damage to the heart in muscular ...
News

Neurologist Urges Greater Focus on Brain Issues in Duchenne
December 19, 2018December 19, 2018
Close to 85 percent of boys and young men with Duchenne muscular dystrophy have ...
columns, You, Me and MD - a Column by Leah Leilani

Here’s How I Decorated My Wheelchair This Christmas
December 18, 2018December 17, 2018
In my family, almost every holiday deserves decorations. Even Valentine’s Day is celebrated, with ...

May 21, 2018May 21, 2018

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

News

The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide Children’s ... Read more

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