Prosensa Enrolls 100th Patient to its Natural History Study of Duchenne Muscular Dystrophy

Leiden, The Netherlands, Nov. 7, 2013 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, today announced the suc­cess­ful enroll­ment of the 100th patient into the Natural History Study of Duchenne mus­cu­lar dys­tro­phy (DMD).
The goal of this obser­va­tion­al study is to char­ac­ter­ize DMD at var­i­ous stages of pro­gres­sion using the same mea­sures used in ongo­ing clin­i­cal stud­ies, such as the “six minute walk test.” No med­ica­tion is being test­ed in this study.
“The enroll­ment of the 100th patient for the Natural History Study rein­forces our com­mit­ment to devel­op­ing inno­v­a­tive treat­ment options for DMD,” said Dr. Giles Campion, Prosensa’s Chief Medical Officer. “This study will fos­ter greater under­stand­ing of the pro­gres­sion of this debil­i­tat­ing dis­ease and will help explore new end­points that could be used to expe­dite drug devel­op­ment.”
Dr. Brenda Wong, Director of the Comprehensive Neuromuscular Center at Cincinnati Children’s and one of the study’s lead inves­ti­ga­tors, adds “this crit­i­cal work on the nat­ur­al his­to­ry of DMD rep­re­sents an impact­ful mile­stone for patients and their fam­i­lies. Our find­ings may enable more effi­cient tri­al design for fol­low-on pro­grams and improved out­comes for patients.”
Patients in the study are assessed every six months for a peri­od of three years to mea­sure their mus­cle strength and func­tion, in addi­tion to how the dis­ease affects their qual­i­ty of life as the con­di­tion evolves over time. Investigators are observ­ing the patients as they per­form var­i­ous phys­i­cal tests, and assess their qual­i­ty of life through sur­vey ques­tions. Furthermore, cer­tain bio­mark­ers are mea­sured through blood and urine sam­ples to inves­ti­gate a pos­si­ble cor­re­la­tion to dis­ease pro­gres­sion.
Prosensa and GlaxoSmithKline (GSK), who are both fund­ing the study, expect to enroll up to 250 DMD patients into the study with any type of muta­tion in the DMD gene between the ages of three and 18. The study is being con­duct­ed in 16 hos­pi­tals across 10 coun­tries with 14 sites already open in the U.S. and Europe. Further infor­ma­tion on the study can be found here: www.ClinicalTrials.gov – Study ID: NCT01753804

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