27 Jul
  • By Nic Williams
  • Cause in

Sarepta’s RNA-targeted technology platforms (PMO and PPMO)

Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, gave a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill provided valuable information on this groundbreaking science. If you were unable to participate, please use the link below to view the recording. Click here to watch the recording of the webinar. You’ll be […]

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12 Jul
  • By Nic Williams
  • Cause in

NS Pharma’s Exon 53 Skipping Program Update

CureDuchenne hosted a webinar with Dr. Paula Clemens and Lauren Morgenroth on the NS Pharma Exon 53 Skipping Program. The webinar was an opportunity for the Duchenne community to learn the most recent status updates, as well as future plans for the science. Click here to watch the recording of the webinar. You’ll be prompted to enter your […]

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22 Jun
  • By Alicia Clough
  • Cause in

Update on Santhera, Idbenone in DMD and SIDEROS Trial

This webinar provided an update from Santhera Pharmaceuticals about the SIDEROS trial of idebenone in boys taking steroids. Santhera also provided an update about their regulatory efforts to bring this treatment to boys with Duchenne in the US and Europe.

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24 Jan
  • By leilanifera
  • Cause in

TRiNDS: A new solution for the Duchenne Research Community

TRiNDS is a specialized contract research organization (CRO) for neuromuscular diseases. It is a full-service CRO with four main pillars of solutions in clinical operations, data management, biostatistics, and study measurements. It is unique with its focus on muscular dystrophies and study outcome developments. It is built on the expertise established with the academic clinical trial network: CINRG. TRiNDS bridges gaps for neuromuscular research by drawing in the key leaders in the field.

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01 Nov
  • By leilanifera
  • Cause in

Questions Regarding Deflazacort Access and Regulatory Status

This informational webinar highlighted the regulatory process to date, updated the community on Marathon Pharmaceutical’s Expanded Access Program (EAP) for deflazacort and discussed what a potential FDA approval of deflazacort would mean in terms of access to the drug in the United States. Some of those who import versions of deflazacort from abroad may have received an update from their distributor with initial details, which was discussed along with steps deflazacort patients might choose to take to help ensure continued access to U.S. supply of deflazacort if FDA approval is granted.

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12 Oct
  • By leilanifera
  • Cause in

Latest Duplication Research Update for Duchenne Webinar

CureDuchenne hosted a webinar with Dr. Kevin Flanigan from Nationwide Children’s Hospital on October 12, 2016. The webinar was an opportunity for the Duchenne community to learn the most recent status of Dr. Flanigan’s duplication mutation research. Dr. Flanigan presented new data on his duplication 2 research.

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