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2019 Duchenne Research News Roundup

2019 Duchenne Research News Roundup

2019 Duchenne Research News Roundup

As 2019 comes to a close, I thought it would be helpful to recap Duchenne research news from this past year. As we reflect on the news from 2019, let us look forward to 2020 with hope.


Nonprofit CureDuchenne is Encouraged to See Gene Editing for Duchenne Advance Through Vertex’s Acquisition of Exonics Therapeutics

CureDuchenne, a nonprofit global leader in research, patient care, and innovation in improving and extending the lives of those living with Duchenne muscular dystrophy (DMD), is enthusiastic with today’s announcement that Exonics Therapeutics, a research company focused on CRISPR/Cas-9 technologies to treat Duchenne muscular dystrophy, has been acquired by Vertex.


Pfizer Presents Initial Clinical Data on Phase 1b Gene Therapy Study for Duchenne Muscular Dystrophy (DMD)

Pfizer Inc. will present initial Phase 1b clinical data on PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy (DMD) at the 25th Annual Parent Project Muscular Dystrophy (PPMD) Connect Conference in Orlando, FL.


Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics

Astellas Pharma Inc. and Audentes Therapeutics, Inc., today announced that they have entered into a definitive agreement for Astellas to acquire Audentes at a price of US$60.00 per share in cash, representing a total equity value of approximately US$3 billion.


Sarepta Therapeutics Announces FDA Approval of VYONDYS 53 (Golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved VYONDYS 53 (golodirsen). VYONDYS 53 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.


Wave Life Sciences Announces Discontinuation of Suvodirsen Development for Duchenne Muscular Dystrophy

Wave Life Sciences Ltd., a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced its decision to discontinue development of Suvodirsen for patients with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping, based on its interim analysis of the Phase 1 open-label extension (OLE) study.


FDA Authorizes First Test to aid in Newborn Screening for Duchenne Muscular Dystrophy

Today, the U.S. Food and Drug Administration authorized marketing of the first test to aid in newborn screening for Duchenne Muscular Dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.


Solid Biosciences Provides Data Update from SGT-001 Development Program

Solid Biosciences Inc. today announced biomarker data from two patients dosed in the second cohort of IGNITE DMD, its Phase I/II study of SGT-001. SGT-001 is the company’s gene transfer candidate under investigation for Duchenne muscular dystrophy (Duchenne).


Clinical Development Program Studying RG6206 (RO7239361) in Duchenne to be Discontinued

We are writing to share with you unfortunate news about our clinical development program studying RG6206 (RO7239361), an investigational anti-myostatin adnectin protein, in ambulatory boys with Duchenne Muscular Dystrophy (DMD).


Mallinckrodt announces the discontinuation of their Phase II clinical trial for MNK-1411

Mallinckrodt has announced the discontinuation of their Phase II clinical trial for MNK-1411, a treatment that aimed to improve muscle health by reducing muscle inflammation and modulating immune system activity.


U.S. FDA Submission of New Drug Application for NS-065/NCNP-01 (Viltolarsen)

Nippon Shinyaku Co., Ltd. announced that it has completed the submission of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for NS-065/NCNP-01 (viltolarsen), which is under development for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.


Santhera Announces Publication of Long-term Idebenone Data from SYROS Study in Duchenne Muscular Dystrophy

Santhera Pharmaceuticals announces publication of the previously reported SYROS study data in prominent peer-reviewed medical journal Neuromuscular Disorders, demonstrating long-term efficacy with idebenone in slowing respiratory function loss in patients with Duchenne muscular dystrophy (DMD) under routine clinical care.


Roche enters licensing agreement with Sarepta Therapeutics to improve the lives of patients living with Duchenne muscular dystrophy

Roche and Sarepta Therapeutics, Inc., today announced the signing of a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States.