New Audentes Patient Advocacy Website Created to Serve Patient Community

AudentesSAN FRANCISCO, CA – February 26, 2015 – Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced its support for Rare Disease Day®, to be held on February 28th, 2015.  The theme of this year’s Rare Disease Day is Living with a Rare Disease.  In honor of the day, Audentes is pleased to announce the launch of the new website, which is dedicated to supporting the patient and caregiver communities with whom Audentes collaborates.

“Audentes is proud to partner with the global rare disease community to support Rare Disease Day 2015,” said Matthew Patterson, President and Chief Executive Officer of Audentes Therapeutics.  “By raising awareness we improve the chances that new treatments are made available for the millions of people who live with these diseases every day.”

In recognition of Rare Disease Day, Audentes employees will be participating in the following activities:

  • Global Genes Project Wear That You Care™/Blue Denim Genes™.  This initiative aims to raise awareness of Rare Diseases and generate funding to support the mission of Global Genes and the communities it serves.
  • Handprints Across America™.  This initiative, which is sponsored by the National Organization for Rare Diseases (NORD), invites individuals and organizations to share a picture and a story about how a rare disease has affected them or had an impact on their lives.
  • An Audentes representative will be attending the Rare Disease Legislative Advocates ( Rare Disease Legislative Conference and other Rare Disease Week events in Washington D.C.

Audentes is also launching the new website to provide patients and caregivers with information related to the diseases that the Company is currently targeting with research and development programs.  The initial focus on X-Linked Myotubular Myopathy (XLMTM) and Pompe disease includes photographs and stories about remarkable individuals living with these serious diseases.  In order to facilitate dialog between Audentes and the patient and caregiver communities, the website will also feature information related to key events, patient and medical meetings, diagnosis and care resources, and activities to develop new therapies for diseases such as XLMTM and Pompe.

About Rare Disease Day

Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face.  It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, a leading independent, non-profit organization committed to the identification, treatment, and cure of rare diseases.

For more information about Rare Disease Day in the U.S., go to  For information about global activities, go to

About Global Genes

Global GenesTM is a leading rare disease patient advocacy organization. The group’s mission is to eliminate the challenges of rare disease by providing patients with educational tools, building awareness, providing critical connections to people and resources, and through investment in technologies that will positively impact affected patients and families. Recognized worldwide by the Blue Denim Genes RibbonTM, Global Genes unites experts, advocates and patients of all ages to stand together in hope for treatments and cures for the estimated 7,000 rare and genetic diseases that impact approximately 35 million Americans and over 350 million people worldwide.

For more information about Global Genes, please visit the following links:


EURORDIS, the European Organization for Rare Diseases, is a non-governmental patient-driven alliance of patient organizations representing over 600 rare disease patient organizations in more than 60 countries. EURORDIS represents the voice of an estimated 30 million people living with a rare disease in Europe. Follow @eurordis or see the EURORDIS Facebook page. For more information visit:

About NORD

Established in 1983, the National Organization for Rare Disorders (NORD) is the primary nonprofit organization representing all patients and families affected by rare diseases in the U.S. It provides programs of advocacy, education, research and patient/family services to improve the lives of all people living with rare diseases. NORD represents more than 200 disease-specific member organizations and partners with many other organizations in specific causes of importance to the rare disease patient community. Follow NORD at and on Twitter at @RareDiseases.

About Audentes Therapeutics, Inc.

Audentes is a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases. The company has two products in development, AT001 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and AT002 for the treatment of Pompe Disease. The company consists of a focused, experienced, and passionate team driven by the goal of improving the lives of patients. Audentes takes pride in strong, global relationships with the patient, research, and medical communities.

For more information regarding Audentes, please visit

About X-Linked Myotubular Myopathy (XLMTM)

X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. It is caused by mutations in the MTM1 gene, which encodes an enzyme called myotubularin. Myotubularin plays an important role in the development and maintenance of muscle cells. XLMTM affects approximately 1 in 50,000 newborn males worldwide.  Audentes is developing AT001 for the treatment of XLMTM in collaboration with Genethon (  AT001 is a novel product based on AAV gene therapy technology.

About Pompe Disease

Pompe Disease is a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It is caused by mutations in a gene that encodes an enzyme called acid alpha-glucosidase (GAA), which is needed by the body to break down glycogen – a stored form of sugar used for energy. Pompe Disease affects approximately 1 in every 40,000 births.  AT002 is a novel product based on AAV gene therapy technology.

Tagged: advocacy, gene therapy, Rare Disease Day