Release Details
PTC Therapeutics Announces Launch of STRIVE Grant Award Program for Cystic Fibrosis
"We are pleased to launch the STRIVE Awards for cystic fibrosis and look forward to the opportunity to support the community in their efforts to serve patients," said
Any patient advocacy organization that serves the CF community and demonstrates the capability to successfully implement the proposed project is eligible to submit an award entry. An external five-member Review Committee will evaluate the feasibility, creativity, budget, impact and sustainability of each proposal. Members of the committee were selected for their understanding of the rare disease community, coupled with skills that include patient advocacy, philanthropy, communications and knowledge of science.
"Cystic fibrosis is a disabling and life-threatening disorder. The organizations serving this community are deeply passionate about their commitment to make a positive difference in the lives of patients and their families. We feel privileged to support these groups and to help make their visions a reality," said
Applications for the 2016 STRIVE Awards for CF are open from
ABOUT CYSTIC FIBROSIS
Cystic fibrosis (CF) is a disabling and life-threatening autosomal recessive disorder resulting from mutations that cause dysfunction in the cystic fibrosis transmembrane conductance regulator (CFTR). In nonsense mutation cystic fibrosis, an interruption in the genetic code - known as a nonsense mutation - prematurely halts the synthesis of CFTR, causing the protein to be short and non-functioning. Nonsense mutations are categorized as Class I mutations that result in little or no production of the CFTR protein. CF patients with Class I mutations typically experience more severe disease symptoms than those with other genotypes, including a shorter life span, a higher probability of end-stage lung disease, and a higher prevalence of pancreatic insufficiency. Approximately 10% of patients have CF due to a Class I nonsense mutation in at least one
allele of the CFTR gene. Available therapies for treatment of lung manifestations of CF, such as inhaled antibiotics, do not address the underlying defect. There are no marketed treatments that target the defect associated with CF caused by nonsense mutations.
ABOUT PTC THERAPEUTICS, INC.:
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company,
please visit our website www.ptcbio.com.
FOR MORE INFORMATION:
Investors:
+1(908) 912-9327
ehill@ptcbio.com
Media:
+1 (908) 912-9551
jomalley@ptcbio.com
Patient Organizations:
+1 (908) 912-9205 or (866) 282-5873
mharmon@ptcbio.com
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