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June 5, 2019June 5, 2019

Santhera Collaborating With Swiss Researchers to Develop New Gene Therapy for LAMA2 MD

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Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of Basel in Switzerland to develop a new gene therapy for treating people ... Read more

June 3, 2019June 4, 2019

‘Serious Concerns’ Raised by Watchdog Group on Cost-effectiveness of DMD Therapies

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The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness of Sarepta‘s exon-skipping therapies Exondys 51 (eteplirsen), and the investigational golodirsen, as ... Read more

May 31, 2019May 31, 2019

Santhera Seeks European Approval of Puldysa to Address Respiratory Problems in DMD

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Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment for respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD). The ... Read more

May 29, 2019May 29, 2019

With Zolgensma’s Approval, Scientists Pursue Similar Gene Therapies in Duchenne

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Now that the U.S. Food and Drug Administration (FDA) has approved Zolgensma — the world’s first gene therapy for spinal muscular atrophy (SMA) — experts say a similar gene ... Read more

May 27, 2019May 27, 2019

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

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RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site ... Read more

May 24, 2019May 24, 2019

FinancialForce Helps Boost Muscular Dystrophy Association’s Fundraising Efforts

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A FinancialForce platform has allowed the Muscular Dystrophy Association (MDA) to enhance fundraising efforts while streamlining accounting activities. Since completing the platform launch and implementing an administrative realignment, the ... Read more

May 22, 2019May 22, 2019

PPMD Awards $100K for Immune Cell Profiling Research for Duchenne and Becker at UCLA

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The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the signature profile of immune cells in patients with Duchenne and ... Read more

May 17, 2019May 17, 2019

Sarepta Acquires Rights to Novel Gene Therapy Candidate to Treat Limb-Girdle MD

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Sarepta Therapeutics is acquiring an investigational gene therapy program focused on calpain-3 for the treatment of limb-girdle muscular dystrophy type 2A (LGMD2A). The candidate therapy was developed by the Research ... Read more

May 15, 2019

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

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With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been ... Read more

May 15, 2019May 15, 2019

Duchenne Teen Hits the Runway at Las Vegas Fashion Show

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Joey Chorzewski has Duchenne muscular dystrophy, a neuromuscular disease that makes him different from most of his peers in Marshfield, Massachusetts. But in other ways — like wanting to ... Read more

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