Updates

March 20, 2019March 20, 2019
New Enhanced Microdystrophin Versions May Help Restore Muscle Function in DMD, Mouse Study Suggests

Engineered small versions of dystrophin protein, known as microdystrophins, have shown the ability to enhance muscle strength and function in preclinical models of Duchenne ... Read more
March 19, 2019March 15, 2019
My Birth Control Injections Have Restored My Quality of Life

The time when a girl’s body matures into that of a young woman isn’t how it’s portrayed in the movies — at least it ... Read more
March 18, 2019March 18, 2019
Firefighters to Hike 130 Miles to Raise Awareness of Neuromuscular Diseases and Promote Newborn Screening

Building on their international trade association’s relationship with the Muscular Dystrophy Association (MDA), two Virginia firefighters will set out on a 130-mile hike this ... Read more
March 15, 2019March 15, 2019
Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD), combines the benefits of two existing therapies — prednisone and eplerenone — on heart and muscle health, but ... Read more
March 14, 2019March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more

News

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 13, 2019March 13, 2019
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ...
News

MDA’s 37th #MDAShamrocks Fundraiser is Underway
March 13, 2019March 13, 2019
To raise awareness and money to back efforts that help those living with muscular ...
columns, You, Me and MD - a Column by Leah Leilani

My First Time Attending the LA Abilities Expo
March 12, 2019March 11, 2019
A few weeks ago, I attended the Los Angeles Abilities Expo for the first time. Because I ...
News

CureDuchenne Sets Calendar for Workshops, Sessions and ‘Futures’ Conference
March 11, 2019March 11, 2019
CureDuchenne, one of the nation’s leading Duchenne muscular dystrophy (DMD) charities, is sponsoring 30 events ...
News

MYO-101 Enables Production of Missing Protein in Children with LGMD2E, Trial Shows
March 8, 2019March 8, 2019
All three children with limb girdle muscular dystrophy type 2E (LGMD2E) treated with the investigational ...
News

Raxone (Idebenone) Shows Promise as DMD Therapy in SYROS Trial
March 6, 2019March 6, 2019
Long-term treatment with Raxone (idebenone) slows down loss of respiratory function in Duchenne muscular ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 6, 2019March 20, 2019
Rare diseases affect about 30 million Americans — roughly the same number as those ...
Join Us in the New Muscular Dystrophy News Forums
March 5, 2019March 4, 2019
I received my mitochondrial myopathy diagnosis 13 years ago. I became involved with the ...
Centogene Collaborates with Sarepta to Identify DMD Patients in the Middle East and North Africa
March 4, 2019March 12, 2019
Edasalonexent May Limit Disease Progression in Boys with Duchenne MD, Phase 1/2 Trial Shows
March 1, 2019March 12, 2019
What Teachers Should Know About Students with Disabilities
February 26, 2019March 12, 2019
New Device Enables Home Monitoring of Respiratory Function in Teens with DMD, Study Reports
February 25, 2019March 8, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 25, 2019March 13, 2019
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ...
Vienna to Host RARE2019 Meeting on Rare Diseases
February 22, 2019February 22, 2019
Edasalonexent Leads to Normal Growth, Other Benefits in Boys with DMD, Phase 1/2 Trial Shows
February 22, 2019March 8, 2019
MDA Releases Duchenne Fact Sheet, Survey Results Pointing to Need for Knowledge of Clinical Trials and Genetic Tests
February 20, 2019February 27, 2019

Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial
February 18, 2019February 22, 2019
Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular ...
Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA
February 15, 2019February 22, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 13, 2019February 20, 2019

News

Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019
February 13, 2019February 20, 2019
With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy ...
columns, You, Me and MD - a Column by Leah Leilani

Tips to Alleviate Symptoms of Constipation
February 12, 2019February 20, 2019
Managing one’s health while living with a disability requires much more than regular trips ...
News

Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces
February 11, 2019February 18, 2019
Capricor Therapeutics announced that patient dosing has resumed in its HOPE-2 trial of CAP-1002, an ...
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 18, 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 ...

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

Subscribe to our mailing list

Pin It on Pinterest