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1、BUILDINGABUSINESSGivingpatientsasay:howtoworkwithpatientadvocacygroupsAnne-LaureWinkler&DavidFinegoldWorkingwithpatientadvocacygroupsposesmanychallengestoentrepreneurs,butthebenefitscanbesubstantial.Here,weexaminetherelationshipbetweenbiotechPTCTherapeuticsandParentProjectMuscular
2、Dystrophytohighlightsomekeyfactorsforsuccessinsuchpartnerships.henachildisdiagnosedwithatermi-PTC124:cysticfibrosis(CF)andDuchennepledgedtoraise$1millionfromparents,andWnaldisease,parentswilltryalmostany-musculardystrophy(DMD).PTCcontributedwithitsinternalresources.thingtohelp.One
3、directioninwhichtheyareSometimeshortlyafter,H.LeeSweeney,Unexpectedly,fourofthefivetargetsyieldedincreasinglychannelingtheireffortsistousechairoftheUniversityofPennsylvaniaSchoolpositiveresults.Thatposedadilemmaforthetheirownmoneytofundearly-stageresearchofMedicine’sdepartmentofph
4、ysiology,partners:eachtargetrepresentedadifferent,atcompanies.ButwhataretheprosandconsintroducedthePTCmanagementteamtocomplementaryapproachtotreatingDMD,yetforentrepreneursofworkingwithinves-representativesofPPMD,agrassrootsorgani-thecostsofpursuingallfourtargetswouldbetorswhohave
5、suchadesperateneedtofindzationfocusedonfindingtreatmentsforDMD.substantial—anestimated$1.7millionforhit-treatments?Welookedatonesuchcollabora-Sweeneyhadbuiltindependentrelationshipsasto-leadeffortsand$15millionforleadopti-tion,ProjectCatalyst,whichisapartnershipascientificadvisort
6、oPTCandascientificdirec-mization.ItwasatthispointthatthepartnersbetweenParentProjectMuscularDystrophytorforPPMDandbroughtthetwotogetherappliedtotheUSNationalInstitutesofHealth(PPMD),agroupofaround3,000parentsandwhenhefeltthetimewasright.ForPPMD’s(NIH)forfunding,whichinJuly2007awar
7、dedrelatives,50ofwhicheachcontributedatleastfounderPatriciaFurlong,wholosttwosonstheresearchcollaborationbetweenSweeney$25,000forresearch,andSouthPlainfield,Newattheagesof15and17toDMD,“itwaslikeaandPTCagrantof$15.4million(seeBox1).Jersey–basedPTCTherapeutics.Basedonourdreamcometru
8、e.”NotonlydidPTC124offerastudyoft
