PTC THERAPEUTICS, INC.
'Local, independent organizations play a vital role in supporting and guiding individuals living with Duchenne and their families as they journey through previously unattainable milestones. We're excited to review the proposals and look forward to the great work the organizations will carry out with the STRIVE grant awards,' said
Until recently, children with Duchenne usually did not live beyond their teens1; however, improvements in the treatment and management of Duchenne have increased life expectancy, and now many Duchenne patients are living well into their 30s and 40s.1,2 As a result, there is an increasing need for psychosocial support for the Duchenne community.3 The future for people living with Duchenne is broadening and innovative programs that provide support and encourage independence are of infinite value.
Since its inception, the STRIVE program has supported more than 35 patient initiatives from not-for-profit organizations from 20 countries. Past STRIVE grant recipients have developed programs to support the transition from adolescence to adulthood, improved access to diagnosis and treatment, supplied vital physical and social care to those affected and their caregivers, created career opportunities for adults with Duchenne and raised awareness of the disease in educational, public, and healthcare settings.
The 2022 application process is now open to patient advocacy organizations. The deadline for submissions is
About the STRIVE Awards Program
PTC launched the Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program in 2015 to support initiatives that benefit the Duchenne community by increasing awareness, diagnosis and education and fostering the development of future patient advocates. Each year, an independent panel of external experts with knowledge in rare diseases, patient advocacy and funding initiatives judges the entries for innovation, vision, and empowerment.
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support and heart complications in their late teens and twenties.
More information regarding Duchenne is available through the
About PTC
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and can globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. Our mission is to provide access to best-in-class treatments for patients who have little to no treatment options. The company's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. We believe this allows us to maximize value for all our stakeholders.
Contact:
Media
T: (908) 912-9406
E: jclemente@ptcbio.com
Patient Organizations
T: (908) 912-9205
T: (866) 282-5873
E: kgalambos@ptcbio.com
References
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3. Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018; 17:251-267.
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