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news-header-image-sarepta

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45

June 26, 2020

06/26/20 8:30 AM EDT — Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne — — Casimersen is the third exon-skipping […]

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NS Pharma - Webinar Header

CureDuchenne Webinar: An Introduction to NS Pharma, Inc. and its access support program, NS Support

June 22, 2020

During this webinar, you will learn about NS Pharma, Inc. (a wholly owned, US subsidiary of Nippon Shinyaku Co., Ltd. based in Kyoto, Japan), focused on developing innovative treatments for […]

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Cure Duchenne News

CureDuchenne Appoints Joanna Kemp to Director of Clinical Programs

June 15, 2020

Newport Beach, CA, June 15, 2020 –­­­  CureDuchenne, a leading global nonprofit focused on funding and finding a cure for Duchenne muscular dystrophy, announced today that Joanna Kemp, BSN, RN, […]

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news-header-image-sarepta

Sarepta Therapeutics Announces Positive Safety and Efficacy Data from the SRP-9001 Micro-Dystrophin Gene Therapy Trial Published in JAMA Neurology

June 15, 2020

06/15/20 11:00 AM EDT — Results at one year demonstrate continued safety and tolerability of SRP-9001 micro-dystrophin gene therapy in four patients with Duchenne muscular dystrophy — — Confirmed vector […]

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EC Grants Orphan Drug Designation to viltolarsen (NS-065/NCNP-01)

June 8, 2020

Kyoto, Japan, June 8, 2020 – Nippon Shinyaku Co., LTD. (Nippon Shinyaku; Kyoto; President, Shigenobu Maekawa) announced that European Commission (EC) has granted Orphan Drug Designation to viltolarsen (NS-065/ NCNP-01) […]

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partner-santhera

Santhera Announces Completion of ReveraGen’s Long-Term Extension Study with Vamorolone in Duchenne Muscular Dystrophy

June 2, 2020

Pratteln, Switzerland, June 2, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces that partner ReveraGen Biopharma Inc. has completed a long-term, open-label extension study of 24 months duration with vamorolone in […]

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partner-santhera

SIDEROS trial community update from Santhera

May 21, 2020

May 20, 2020 Dear Duchenne community, As you know from our previous updates, Santhera’s main focus in the U.S. in the first half of the year has been to bring […]

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partner-pfizer

PFIZER’S NEW PHASE 1B RESULTS OF GENE THERAPY IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY (DMD) SUPPORT ADVANCEMENT INTO PIVOTAL PHASE 3 STUDY

May 15, 2020

Friday, May 15, 2020 – 8:00amEDT Pfizer Inc. (NYSE: PFE) today announced updated Phase 1b clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy […]

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PFIZER-NEWS-HEADER

CureDuchenne Applauds Pfizer’s Encouraging Data from Phase Ib Mini-Dystrophin Gene Therapy Trial for Duchenne Muscular Dystrophy

May 15, 2020

Newport Beach, CA, May 15, 2020 –­­­  CureDuchenne, a leading global nonprofit focused on funding and finding a cure for Duchenne muscular dystrophy, congratulates Pfizer for announcing data from subjects […]

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