Audentes Therapeutics Recognizes Rare Disease Day by Launching "Be Bold for Courageous Patients" Program to Support Patients Living with Rare Diseases

SAN FRANCISCO, Feb. 28, 2019 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today announced its support for Rare Disease Day^® with the launch of "Be Bold for Courageous Patients," a program designed to meaningfully impact the lives of patients living with rare diseases.

"Be Bold for Courageous Patients" is intended to enhance and expand the Company's commitment to rare disease patients and their families.  As part of this year's effort, Audentes will provide a sponsorship grant to help support children from the rare disease community to attend The Painted Turtle and Double H Ranch, medical specialty camps that offer the opportunity for kids to enjoy being kids in a camp environment specifically built for their needs.  Children and their families will attend The Painted Turtle during Neuromuscular and Genetic Family Weekend from March 1 – 3, in Lake Hughes, California, or Double H Ranch during Camp Inspiration for children who are BIPAP or ventilator dependent from June 7 – 11, in Lake Luzerne, New York.

These unique camps offer children and their families the opportunity to sing, laugh, grow, and discover their potential.  Audentes employees are also volunteering their time to make camper family weekend kits, blankets, and turtle pillows for families attending the Neuromuscular and Genetic Family Weekend and Camp Inspiration.  Both The Painted Turtle and Double H Ranch are part of SeriousFun Children's Network founded by Hollywood actor and philanthropist Paul Newman.  All SeriousFun camps and programs are provided at no cost to families and rely on grants and donations to help them achieve their mission.  Families can apply for an opportunity to attend directly through each camp's website and by following their standard application process.

"Audentes is derived from the Latin word 'audeo,' which means 'those who have courage; those who have boldness, daring.'  The name Audentes reflects our heritage as a patient-focused company dedicated to making a bold effort to find solutions for patients," said Matthew R. Patterson, Chairman and Chief Executive Officer.  "To that end, we are pleased to launch 'Be Bold for Courageous Patients' to further serve the needs of the rare disease community in unique and positive ways, including teaming up with The Painted Turtle and Double H Ranch.  Championing and serving patient communities creates a sense of mission and purpose that inspires all Audentes employees, and our broader commitment to the rare disease community is core to our company beliefs."

"We are excited to have Audentes as a sponsoring partner this year," said Chris Butler, CEO of The Painted Turtle.  "This meaningful support enables children living with rare diseases to have a positive camp experience and help their families bond through our Family Weekend programs."

The Audentes "Be Bold for Courageous Patients" program is comprised of the following initiatives:

• Support rare disease work. Provide corporate grants and contributions that aid the work of scientists, researchers, medical professionals, and advocacy groups that are finding ways to help the rare disease community and making a lasting, positive impact. In 2019, Audentes will contribute to more than 30 organizations.
• Impact the patient community in a unique, positive way. Beyond research and development of innovative gene therapy products, Audentes wants to positively impact the lives of patients and families in a unique way, such as supporting The Painted Turtle and Double H Ranch medical specialty camps.
• Evolve employee volunteerism companywide to further Audentes' commitment to the patient community. All employees receive time off to volunteer for the organizations that most inspire them, focusing on the patient community.

There are approximately 7,000 different types of rare diseases affecting up to 350 million people worldwide.  Eighty percent of rare diseases are genetic in origin, and thus are present throughout a person's life, even if symptoms do not immediately appear.  An estimated 50 percent of the people affected by a rare disease are children, and 30 percent of children with rare disease will not live to see their fifth birthday.¹  Although rare disease patients and their families face many challenges, enormous progress is being made every day.

About Rare Disease Day
Rare Disease Day takes place on the last day of February, a month known for having a 'rare' number of days.  It was first launched in Europe by EURORDIS and its Council of National Alliances in 2008 and is now observed in more than 94 countries.  Rare Disease Day is sponsored in the United States by the National Organization for Rare Disorders (NORD)^®, a leading independent, non-profit organization committed to the identification, treatment, and cure of rare diseases.

About The Painted Turtle
The Painted Turtle's mission is to provide a year-round, life-changing and authentic camp experience for children with chronic and life-threatening illnesses. The Painted Turtle supports children's medical needs, inspires them to reach beyond their illness, and provides care, education, and respite for their families. All campers and families attend free of charge.  For more information, visit www.thepaintedturtle.org.

About the Double H Ranch
The Double H Ranch, co-founded by Charles R. Wood and Paul Newman, provides specialized programs and year-round support for children and their families dealing with life-threatening illnesses.  Our purpose is to enrich their lives and provide camp experiences that are memorable, exciting, fun, empowering, physically safe and medically sound.  All programs are FREE of charge and capture the magic of the Adirondacks.  For more information, visit www.doublehranch.org. 

About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases.  We are currently conducting Phase 1/2 clinical studies of our lead product candidates, AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar syndrome.  We have two additional product candidates in development, including AT845 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT).  We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.

For more information regarding Audentes, please visit www.audentestx.com.

Audentes Contacts:

Investor Contact:
Andrew Chang
415.818.1033
[email protected] 

Media Contact:
Katie Hogan
415.951.3398
[email protected]

¹ https://globalgenes.org/rare-diseases-facts-statistics/

SOURCE Audentes Therapeutics, Inc.

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