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June 19, 2019June 19, 2019

YourDNA and Cure Rare Disease Collaborate to Raise DMD Awareness, Advance Genomics

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To help make cutting-edge genetic research more accessible, and raise awareness of rare diseases such as Duchenne muscular dystrophy (DMD), YourDNA and Cure Rare Disease (CRD) are joining forces. The ... Read more

June 17, 2019

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

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People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most ... Read more

June 17, 2019June 17, 2019

PTC Therapeutics Gets FDA Approval for Emflaza for Ages 2-5

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Initially approved in the United States for Duchenne muscular dystrophy (DMD) patients age 5 and older, the corticosteroid Emflaza is now available for those as young as 2. The ... Read more

June 14, 2019June 14, 2019

DMD Patients Are Living Longer, While New Cases Hold Steady, Study Finds

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The prevalence rate of Duchenne muscular dystrophy (DMD) in the United States has increased, largely due to better treatments that are helping patients live longer, while the number of ... Read more

June 12, 2019June 12, 2019

Blocking Protein That Hinders Muscle Growth Possible Way of Treating LGMD1, Mouse Study Suggests

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Lithium chloride — once used as table salt — improved muscle size and strength in mouse models of limb-girdle muscular dystrophy D1 (LGMD1) by blocking a protein that is ... Read more

June 10, 2019

Vertex Acquires Exonics Therapeutics, Advancing Gene-editing Treatment for DMD

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Biotechnology company Vertex has acquired Exonics Therapeutics, which focuses on using gene-editing technologies to treat Duchenne muscular dystrophy (DMD) — a move that is being hailed as a positive ... Read more

June 7, 2019June 7, 2019

Researchers Awarded $175K Grant from Parent Project Muscular Dystrophy to Optimize CRISPR/Cas9 for DMD Gene Therapy

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Researchers at the University of California in Los Angeles (UCLA) have been awarded a $175,000 grant from Parent Project Muscular Dystrophy (PPMD) to make CRISPR/Cas9 as safe and effective as possible as a gene ... Read more

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

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