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January 18, 2019January 18, 2019

Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program

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The U.S. Food and Drug Administration (FDA) selected Wave Life Sciences‘ Phase 2/3 trial assessing the efficacy and safety of WVE-210201 (suvodirsen) in Duchenne muscular dystrophy for its pilot program for complex innovative ... Read more

January 16, 2019January 16, 2019

Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD

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Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Aldevron, which produces biological ... Read more

January 14, 2019January 14, 2019

DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK

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Until recently, the only two hospitals in the U.K. with the expertise to run Duchenne muscular dystrophy (DMD) clinical trials were turning studies away — they didn’t have the ... Read more

January 11, 2019January 11, 2019

PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies

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Kanneboyina Nagaraju, PhD, and his research team at Binghamton University, State University of New York will get a $329,000 Parent Project Muscular Dystrophy (PPMD) grant to study responses to ... Read more

January 9, 2019January 9, 2019

Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow

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A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys with Duchenne muscular dystrophy (DMD) at multiple sites in the U.S. and will soon open ... Read more

January 7, 2019January 7, 2019

Capricor Places Hold on Phase 2 Trial of Cell Therapy After Patient Has Allergic Reaction

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Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne muscular dystrophy (DMD) investigative cell therapy CAP-1002 after a patient experienced a severe allergic reaction during blind ... Read more

January 4, 2019January 4, 2019

Myonexus’ MYO-102 Earns FDA’s Orphan Drug Status for LGMD 2D

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The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ MYO-102, a gene therapy for limb-girdle muscular dystrophy type 2D (LGMD 2D), the company announced. The ... Read more

January 4, 2019January 4, 2019

Project Hercules Enlists Industry’s Help to Make Duchenne Therapies Affordable

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Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the ... Read more

January 2, 2019January 2, 2019

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

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Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program

Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD

DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK

PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies

Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow

Capricor Places Hold on Phase 2 Trial of Cell Therapy After Patient Has Allergic Reaction

Myonexus’ MYO-102 Earns FDA’s Orphan Drug Status for LGMD 2D

Project Hercules Enlists Industry’s Help to Make Duchenne Therapies Affordable

Top 10 Muscular Dystrophy Articles of 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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