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August 2, 2019August 2, 2019

Independent Review Board Clears HOPE-2 Trial to Continue Testing CAP-1002 for DMD

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The ongoing Phase 2 HOPE-2 study has been cleared to continue exploring the potential of CAP-1002 therapy in boys and young men with advanced Duchenne muscular dystrophy (DMD). An ... Read more

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

July 31, 2019July 31, 2019

November Ascent of Nepal’s Mt. Everest to Raise Money for Duchenne Research

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Climbing up the world’s tallest mountain isn’t for everyone. With that in mind, a small New England-based charity plans just such an adventure this November — for the third ... Read more

July 31, 2019July 31, 2019

Invitae Offering Free Genetic Testing for Muscular Dystrophy to Those Eligible in US and Canada

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Invitae has opened a program offering free genetic testing and post-test counseling to people suspected of having diseases that include muscular dystrophy (MD) under a partnership with pharmaceutical firms. The goal is ... Read more

July 29, 2019July 29, 2019

Father Inspired by Son to Spearhead Duchenne Parent Project Italy

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Filippo Buccella, an Italian pharmacist from Rome, clearly remembers the day in 1993 when he brought his nearly 3-year-old son, Luca, to the hospital. Luca was about to undergo ... Read more

July 26, 2019July 26, 2019

DMD Expert Advises Parents: ‘How to Talk to School, Family and Kids About Duchenne’

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If there’s anyone who knows a thing or two about how to discuss Duchenne muscular dystrophy (DMD) with your son, his siblings, his teachers and his friends, it’s James ... Read more

July 24, 2019July 26, 2019

As PPMD Turns 25, Founder Pat Furlong Reflects on Advances in Duchenne

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Back in June 1984, a doctor calmly informed Pat Furlong that her sons — 4-year-old Chris and 6-year-old Patrick — had Duchenne muscular dystrophy. It was the first time she ... Read more

July 22, 2019July 22, 2019

MDA Awards $389K to UCLA Scientist to Develop Potential DMD Treatment Targeting Sarcospan

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The Muscular Dystrophy Association (MDA) awarded $389,000 in funding to a researcher from University of California, Los Angeles (UCLA) to support the development of a small molecule that holds ... Read more

July 19, 2019

European Initiative Targets Diagnosis, Treatment of Rare Diseases

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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

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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

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