March 19, 2019March 15, 2019
My Birth Control Injections Have Restored My Quality of Life
columns, You, Me and MD - a Column by Leah Leilani
The time when a girl’s body matures into that of a young woman isn’t how it’s portrayed in the ... Read more
March 18, 2019March 18, 2019
Firefighters to Hike 130 Miles to Raise Awareness of Neuromuscular Diseases and Promote Newborn Screening
News
Building on their international trade association’s relationship with the Muscular Dystrophy Association (MDA), two Virginia firefighters will set out ... Read more
March 15, 2019March 15, 2019
Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds
News
Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD), combines the benefits of two existing therapies — prednisone and eplerenone — on ... Read more
March 14, 2019March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
News
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves ... Read more
March 13, 2019March 13, 2019
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
News
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies ... Read more
March 13, 2019March 13, 2019
MDA’s 37th #MDAShamrocks Fundraiser is Underway
News
To raise awareness and money to back efforts that help those living with muscular dystrophy (MD), the Muscular ... Read more
March 12, 2019March 11, 2019
My First Time Attending the LA Abilities Expo
columns, You, Me and MD - a Column by Leah Leilani
A few weeks ago, I attended the Los Angeles Abilities Expo for the first time. Because I am now a moderator for ... Read more
March 11, 2019March 11, 2019
CureDuchenne Sets Calendar for Workshops, Sessions and ‘Futures’ Conference
News
CureDuchenne, one of the nation’s leading Duchenne muscular dystrophy (DMD) charities, is sponsoring 30 events across the U.S. this year ... Read more
March 8, 2019March 8, 2019
MYO-101 Enables Production of Missing Protein in Children with LGMD2E, Trial Shows
News
All three children with limb girdle muscular dystrophy type 2E (LGMD2E) treated with the investigational gene therapy MYO-101 in a ... Read more
March 6, 2019March 6, 2019
Raxone (Idebenone) Shows Promise as DMD Therapy in SYROS Trial
News
Long-term treatment with Raxone (idebenone) slows down loss of respiratory function in Duchenne muscular dystrophy (DMD) patients for up to ... Read more
March 6, 2019March 6, 2019
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
News
Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet ... Read more
March 5, 2019March 4, 2019
Join Us in the New Muscular Dystrophy News Forums
columns, You, Me and MD - a Column by Leah Leilani
I received my mitochondrial myopathy diagnosis 13 years ago. I became involved with the Muscular Dystrophy Association (MDA) soon ... Read more

March 19, 2019March 15, 2019

My Birth Control Injections Have Restored My Quality of Life

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

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 wasn’t for me. I didn’t ... Read more

March 18, 2019March 18, 2019

Firefighters to Hike 130 Miles to Raise Awareness of Neuromuscular Diseases and Promote Newborn Screening

News

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 month to raise awareness of ... Read more

March 15, 2019March 15, 2019

Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

News

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 with less detrimental side effects, ... Read more

March 14, 2019March 14, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

News

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 scheduled, at the end of ... Read more

March 13, 2019March 13, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

March 13, 2019March 13, 2019

MDA’s 37th #MDAShamrocks Fundraiser is Underway

News

To raise awareness and money to back efforts that help those living with muscular dystrophy (MD), the Muscular Dystrophy Association (MDA) has launched its venerable #MDAShamrocks campaign. More than ... Read more

March 12, 2019March 11, 2019

My First Time Attending the LA Abilities Expo

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

A few weeks ago, I attended the Los Angeles Abilities Expo for the first time. Because I am now a moderator for the Muscular Dystrophy News Forums, I thought it would be ... Read more

March 11, 2019March 11, 2019

CureDuchenne Sets Calendar for Workshops, Sessions and ‘Futures’ Conference

News

CureDuchenne, one of the nation’s leading Duchenne muscular dystrophy (DMD) charities, is sponsoring 30 events across the U.S. this year to educate patients and their families about all aspects of ... Read more

March 8, 2019March 8, 2019

MYO-101 Enables Production of Missing Protein in Children with LGMD2E, Trial Shows

News

All three children with limb girdle muscular dystrophy type 2E (LGMD2E) treated with the investigational gene therapy MYO-101 in a clinical trial showed marked production of beta-sarcoglycan, the missing protein ... Read more

March 6, 2019March 6, 2019

Raxone (Idebenone) Shows Promise as DMD Therapy in SYROS Trial

News

Long-term treatment with Raxone (idebenone) slows down loss of respiratory function in Duchenne muscular dystrophy (DMD) patients for up to six years, according to recent results from Santhera Pharmaceuticals’ SYROS trial. ... Read more

March 6, 2019March 6, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

News

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known ... Read more

March 5, 2019March 4, 2019

Join Us in the New Muscular Dystrophy News Forums

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

I received my mitochondrial myopathy diagnosis 13 years ago. I became involved with the Muscular Dystrophy Association (MDA) soon afterward. The organization is known for its Labor Day telethons previously ... Read more