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Catabasis Shares March 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter, including information about edasalonexent and COVID-19, and responses to questions they have received. Click here to download the PDF.

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NS Pharma Announces Marketing Authorization in Japan of VILTEPSO® for the Treatment of Duchenne

Good news out of Japan! NS Pharma, Inc., announced that their parent company, Nippon Shinyaku Co., Ltd., has received approval for viltolarsen in Japan for people with Duchenne amenable to exon 53 skipping therapy. Viltolarsen…

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NS Pharma Shares Community Letter Regarding COVID-19

NS Pharma, Inc. has shared an update with the community responding to questions about how COVID-19 may be affecting ongoing studies of viltolarsen, the recently launched Expanded Access Program (EAP) and review of the viltolarsen…

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ReveraGen BioPharma Shares Community Update on Vamorolone Trials and COVID-19

ReveraGen has shared an update with the community regarding the Vamorolone trials and COVID-19. Read the Community Letter from ReveraGen Dear Duchenne families, As we are all confronted with the uncertainty of Covid-19, the team…

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Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

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Catabasis Shares Update on Edasalonexent and COVID-19

Catabasis Pharmaceuticals has shared an update responding to questions about edasalonexent and COVID-19. To date, Catabasis has not identified any safety concerns related to COVID-19, and they continue to monitor the trial for the safety…

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PTC Shares Update on EMFLAZA Availability During Coronavirus

PTC Therapeutics has shared a statement on EMFLAZA availability during the coronavirus (COVID-19) crisis, stating that the supply of EMFLAZA will not be affected by the coronavirus (COVID-19). PTC reports that they are closely monitoring…

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Solid Biosciences Provides Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD…

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NS Pharma Announces Launch of Expanded Access Program for Viltolarsen

NS Pharma, Inc. has announced the launch of the viltolarsen Expanded Access Program in the United States for patients with Duchenne muscular dystrophy who are amenable to exon 53 skipping therapy and meet other eligibility…

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WATCH: Teen & Young Adult Trial Participation in Duchenne – Industry & Community Perspectives – Webinar Recording

On Wednesday, March 4, 2020, Santhera Pharmaceuticals joined PPMD for a webinar to discuss teen and young adult clinical trial participation. We heard from Santhera about lessons learned by conducting one of the largest trials…

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