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September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products ... Read more

September 21, 2018September 21, 2018

Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A

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Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type ... Read more

September 19, 2018September 19, 2018

Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

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Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 ... Read more

September 17, 2018September 17, 2018

Obsessive Compulsive Disorders Evident in Duchenne Patients and in Need of Treatment, Study Says

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Obsessive compulsive disorder (OCD), a type of “internalizing disorder,” are evident in children with Duchenne muscular dystrophy, and particularly associated with anxiety and places considerable stress on the patient’s family, ... Read more

September 14, 2018September 14, 2018

Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports

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Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with Duchenne muscular dystrophy (DMD). The study with that finding, “Gene editing ... Read more

September 12, 2018September 12, 2018

STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide

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PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which recognize the efforts of nonprofit organizations working to serve the ... Read more

September 10, 2018September 10, 2018

Numotion, Sing Me a Story Partner to Help Children with Neuromuscular Diseases Turn Life Stories to Songs

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Nonprofit Sing Me a Story and Numotion are collaborating to help children with various neuromuscular diseases served by the Muscular Dystrophy Association (MDA) share their unique stories through song. This alliance ... Read more

September 7, 2018September 7, 2018

Pfizer Stops Phase 2 Clinical Studies of Domagrozumab in DMD Boys

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Pfizer is halting the clinical development of domagrozumab (PF-06252616) as treatment for Duchenne muscular dystrophy (DMD) in an ongoing Phase 2 trial. The latest trial data failed to demonstrate ... Read more

September 5, 2018September 5, 2018

PPMD and Jett Foundation Among Groups Gearing Up for Sept. 7, World Duchenne Awareness Day

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With an emphasis on standards of care, groups such as the Parent Project Muscular Dystrophy and the Jett Foundation are gearing up for Sept. 7, World Duchenne Awareness Day ... Read more

August 31, 2018August 31, 2018

FSH Society Holding ‘Walk & Roll’ Event in Pacific Northwest to Raise Funds, Awareness for FSHD

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The Pacific Northwest chapter of the FSH Society is holding its first-ever “Walk & Roll to Cure FSHD” event Sept. 22 in Puyallup, Washington, a small city about 35 miles south ... Read more

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