Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12

PTC To Test RG7800 in SMA

PTC To Test RG7800 in SMA

The investigational compound is designed to raise levels of the SMN protein, a lack of which is the underlying cause of spinal muscular atrophy (SMA)

BMD: Follistatin Gene Transfer Results Encouraging

BMD: Follistatin Gene Transfer Results Encouraging

Four of six men with Becker muscular dystrophy increased their six-minute walking distance after injections of follistatin genes into the thigh muscles of both legs

BioBlast Is Developing an OPMD Drug

BioBlast Is Developing an OPMD Drug

Israeli biotechnology company BioBlast is developing and testing Cabaletta, designed to counteract abnormal clumping of cellular proteins in oculopharnyngeal muscular dystrophy (OPMD)

AveXis SMA Gene Therapy Trial Continues Recruiting

AveXis SMA Gene Therapy Trial Continues Recruiting

The first three infants in a gene therapy trial for spinal muscular atrophy have been treated; the trial continues, and additional trials are planned

Drug Development for DMD: Fall 2014 Update

Drug Development for DMD: Fall 2014 Update

Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline

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Dedicated MDA family caregivers share best practices for supporting loved ones and themselves

posted on October 30, 2014 - 9:19am
“If I was down, who was going to take care of him?” 

Women with neuromuscular disorders need the same gynecologic care as other women — and sometimes more

posted on October 30, 2014 - 9:17am
Katrina Gossett knows the importance of gynecologic (pelvic) exams, but she is not eager to repeat her last exam experience. Katrina Gossett, 29, has only had one gynecologic (pelvic) exam in her life, and it’s not an experience she’s eager to repeat.

Through its fundraising, advocacy, health care services and support network, MDA helps make possible the everyday successes of its families and the lifesaving work of its researchers

posted on October 30, 2014 - 9:15am
For people fighting neuromuscular disease,“progress” is a word that describes encouraging advances in the search for treatments and cures. But it’s also a word that evokes the forward-looking perspectives and remarkable life journeys of those living with these diseases who meet extraordinary challenges.  

After the fear subsided, my diagnosis gave me a healthy perspective on life

posted on October 30, 2014 - 9:12am
Upon hearing my diagnosis — type 1 myotonic muscular dystrophy (MMD, or DM) — at the age of 20, I must admit, my world crumbled for a moment. I’ll never forget how the room seemed to shrink and spin, how the doctor’s voice morphed into white noise, and how visions of immobility caught my breath. 

They say 'three's a crowd,' but what if the third person is your personal care assistant? A psychologist with SMA offers some dating strategies.

posted on October 30, 2014 - 9:11am
“Samantha” is a typical 30-something single female who has a successful, fulfilling career in investment banking. Samantha also enjoys an active social life, but when she looks around her and sees friends and peers in romantic relationships, she can’t help but desire the same for herself.

Meet the Marquez family

posted on October 30, 2014 - 9:09am
Earlier this year, Bonnie and Ethan Marquez and their sons, Peyton, 8, and Logan, 11, were on vacation in Yosemite National Park when Peyton, who has Duchenne muscular dystrophy (DMD), became intrigued by the park’s famous Half Dome — a 4,700-foot-high granite rock formation in the shape of, you guess it, a dome sliced in half.