13 Jun
  • By L Fera
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Bamboo Therapeutics – Developing a novel gene therapy for patients with Duchenne muscular dystrophy

Bamboo Therapeutics is a recently formed biotechnology company focused on advancing therapies for rare pediatric diseases of the central and neuromuscular systems. Bamboo was founded to advance the pioneering laboratory work of Dr. Jude Samulski, PhD, the former Director of the Gene Therapy Center at the University of North Carolina into human clinical trials. “Gene Therapy” describes the idea of introducing new genetic material into cells to correct an existing problem, often due to a disease-causing mutation. During the June 13, 2016 webinar, Dr. Samulski explained the basics of gene therapy and described Bamboo’s approach to developing a treatment for Duchenne; by utilizing a disabled virus to deliver a small, highly optimized version of the dystrophin gene systemically to skeletal and cardiac muscle.

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03 Nov
  • By L Fera
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HOPE-Duchenne: A Clinical Trial for Individuals with Heart Disease Related to DMD

Capricor Therapeutics and CureDuchenne hosted a webinar on November 3, 2015. The presentation discussed the HOPE-DUCHENNE Trial. The U.S. Food and Drug Adminstration has cleared Capricor’s Investigational New Drug Application for the clinical development of CAP-1002 for the treatment of patients with Duchenne muscular dystrophy. The webinar provided an overview of the clinical trial which is being conducted at several sites in the U.S.

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19 Jun
  • By L Fera
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Capricor Therapeutics Research Update for Duchenne

Capricor Therapeutics and CureDuchenne hosted a webinar on June 19, 2015. The presentation discussed the HOPE-DUCHENNE Trial. The U.S. Food and Drug Administration has cleared Capricor’s Investigational New Drug Application for the clinical development of CAP-1002 for the treatment of patients with Duchenne muscular dystrophy.

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21 Nov
  • By L Fera
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A New Duplication Exon 2 Mouse Model:A Tool for Studies of Duplication Skipping for Duchenne

CureDuchenne was pleased to host the “New Duplication Exon 2 Mouse Model: A Tool for Studies of Duplication Skipping for Duchenne” a webinar with Dr. Kevin Flanigan on November 21, 2013. The webinar was an opportunity for parents to learn about the duplication mutation research and an exciting exon skipping method using AAV delivery with U7 from Dr. Kevin Flanigan at Nationwide Children’s Hospital. CureDuchenne has provided the lead funding for Dr. Flanigan’s duplication research.

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13 Sep
  • By L Fera
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Drug Development and Clinical Trial Participation Webinar

CureDuchenne was pleased to host the “Drug Development and Clinical Trial Participation – A Webinar for the Rare Disease Community” on Friday, September 13, 2013. The webinar was an opportunity for parents to learn about the drug development process – from the pre-clinical phase to the clinical phase to the post approval phase and the regulatory process guiding these phases – from industry experts.

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20 Feb
  • By L Fera
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February 2013 FDA Webinar

CureDuchenne was honored to host a webinar on February 20, 2013, in collaboration with PPMD and MDA, to discuss accelerated approval for drugs for rare diseases. Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research, presented detailed information on the different options available to speed the […]

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