July 19, 2019
European Initiative Targets Diagnosis, Treatment of Rare Diseases
News
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the ... Read more
July 19, 2019July 19, 2019
Cell Therapy CAP-1002 Improves Muscle, Lung Function in Boys with DMD, Interim Data Show
News
CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and lung function in ... Read more
July 18, 2019July 16, 2019
‘Disabled’ vs. ‘Having a Disability’: Debating the Power of Words
columns, Living, Learning, Thriving - a Column by Ralph Yaniz
In the disability community, which is as diverse and broad as any other, there is a debate over terminology. ... Read more
July 17, 2019July 17, 2019
Pfizer’s Gene Therapy Holds Promise in Treatment of DMD, Early Data Show
News
PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy (DMD), shows promising ... Read more
July 15, 2019July 17, 2019
UberWAV Is the Next Best Thing for Accessible Transportation
columns, Hawk's-Eye View - a Column by Hawken Miller
I discovered something different about Washington, D.C. when I moved there for the summer: wheelchair-accessible Ubers are everywhere. Los ... Read more
July 15, 2019July 15, 2019
Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows
News
Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more ... Read more
July 12, 2019July 17, 2019
$500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy
News
For more than 20 years, Robert (Bob) Bennett was a steadfast leader and supporter of the Muscular Dystrophy Association ... Read more
July 11, 2019July 9, 2019
The Speak Foundation Speaks Loudly: A Prelude to September in Chicago
columns, Living, Learning, Thriving - a Column by Ralph Yaniz
I recently attended The Speak Foundation’s 11th Annual Conference. This event is for individuals with neuromuscular diseases and their ... Read more
July 10, 2019
Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
News
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were ... Read more
July 10, 2019July 10, 2019
Potential DMD Treatment, Pamrevlumab, Helps to Preserve Lung, Heart and Upper Limb Function in Youth, Early Trial Data Show
News
Early trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or even improve, ... Read more
July 8, 2019July 2, 2019
Here’s How I Explain My Experience with Duchenne to Others
columns, Hawk's-Eye View - a Column by Hawken Miller
Duchenne muscular dystrophy is not easy for people to wrap their heads around. Most haven’t heard of the disease, ... Read more
July 8, 2019July 8, 2019
MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy
News
The Muscular Dystrophy Association (MDA) has awarded Locana $550,000 through its MDA Venture Philanthropy (MVP) program to help advance the biotech ... Read more

July 19, 2019

European Initiative Targets Diagnosis, Treatment of Rare Diseases

News

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of ... Read more

July 19, 2019July 19, 2019

Cell Therapy CAP-1002 Improves Muscle, Lung Function in Boys with DMD, Interim Data Show

News

CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and lung function in a group of boys and young men at advanced stages ... Read more

July 18, 2019July 16, 2019

‘Disabled’ vs. ‘Having a Disability’: Debating the Power of Words

columns, Living, Learning, Thriving - a Column by Ralph Yaniz

In the disability community, which is as diverse and broad as any other, there is a debate over terminology. I have seen this play out on my Twitter account ... Read more

July 17, 2019July 17, 2019

Pfizer’s Gene Therapy Holds Promise in Treatment of DMD, Early Data Show

News

PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy (DMD), shows promising safety and efficacy results in a small group of boys with DMD, ... Read more

July 15, 2019July 17, 2019

UberWAV Is the Next Best Thing for Accessible Transportation

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

I discovered something different about Washington, D.C. when I moved there for the summer: wheelchair-accessible Ubers are everywhere. Los Angeles, where I come from, has a dearth of accessible ... Read more

July 15, 2019July 15, 2019

Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows

News

Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more advanced disease, compared to standard glucocorticoid therapy, an analysis of clinical trials ... Read more

July 12, 2019July 17, 2019

$500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy

News

For more than 20 years, Robert (Bob) Bennett was a steadfast leader and supporter of the Muscular Dystrophy Association (MDA). To honor his legacy, the Robert M. & Marjie ... Read more

July 11, 2019July 9, 2019

The Speak Foundation Speaks Loudly: A Prelude to September in Chicago

columns, Living, Learning, Thriving - a Column by Ralph Yaniz

I recently attended The Speak Foundation’s 11th Annual Conference. This event is for individuals with neuromuscular diseases and their families. We met in the beautiful city of Atlanta, for ... Read more

July 10, 2019

Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients

News

The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the ... Read more

July 10, 2019July 10, 2019

Potential DMD Treatment, Pamrevlumab, Helps to Preserve Lung, Heart and Upper Limb Function in Youth, Early Trial Data Show

News

Early trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or even improve, lung and heart function in non-ambulatory youth with Duchenne muscular dystrophy ... Read more

July 8, 2019July 2, 2019

Here’s How I Explain My Experience with Duchenne to Others

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

Duchenne muscular dystrophy is not easy for people to wrap their heads around. Most haven’t heard of the disease, which is rare and affects about 1.02 males ages 5 ... Read more

July 8, 2019July 8, 2019

MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy

News

The Muscular Dystrophy Association (MDA) has awarded Locana $550,000 through its MDA Venture Philanthropy (MVP) program to help advance the biotech company’s therapy platform for myotonic dystrophy (DM). Specifically, the grant ... Read more