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1、201R1eportORPHANDRUGSINDEVELOPMENTFORRareDiseasesPRESENTEDBYAMERICA’SBIOPHARMACEUTICALRESEARCHCOMPANIESMoreThan400MedicinesinDevelopmenttoTreatorPreventRareDiseasessweissueourreportonrarediseases,Ican’thelpbutORPHANDRUGSINDEVELOPMENT*thinkofthewordsofamotherwhosedaug
2、hterhasAutoimmuneDisorders18ALennox-GastautSyndrome,whichcausesmultiple,dailyseizures.Inadocumentaryaboutthedisease,shegivesBloodDisorders12thefollowingadvicetootherparents:“Don’ttrylookingatCancer107tomorrow.Don’teventhinkofthenexthour.JustlookfortheCancer,Blood79ne
3、xtminute.Andifthenextminuteistoofaraway,lookfortheCancer,Skin31nextsecond.We’vehadtodothisattimes.Butthereishope.”Cancer-RelatedConditions10CardiovascularDiseases6Rememberingher,IwasgratifiedtofindthatamongtheEyeDisorders11460medicinesindevelopmentforrarediseases,one
4、isforGastrointestinalDisorders10Lennox-GastautSyndrome.Withgreatscience,andsomegoodfortune,wehopeitwillproveasolution.Meanwhile,GeneticDisorders67muchworkremains.GrowthDisorders5InfectiousDiseases31Rarediseases,takentogether,aren’trareatall.Infact,NeurologicalDisorde
5、rs37accordingtotheNationalInstitutesofHealth,25-30millionRespiratoryDisorders14Americanshaveoneofthenearly7,000diseasesthatareTransplantation20officiallydeemed“rare”becausealonetheyeachaffectOther37fewerthan200,000people.Sometimes,onlyafewhundredAmericansareknowntoha
6、veaparticularraredisease.*Somemedicinesarelistedinmorethanonecategory.Simplygettingadiagnosisoftenbecomesaquest,sinceaAmerica’sbiopharmaceuticalresearchcompaniesarelongseriesofdoctorsmayhaveneverheardoforseenthecontinuingthatprogress.The460medicinesforraredisease.Som
7、etimes,patientsandparentsofasickchildarediseasesinthisreportareallinlaterstagesofthepipeline,initiallyrelievedwhentoldtheyortheirchildhasadiseasemeaninginclinicaltrialsorunderreviewbytheFDA.they’veneverheardof,becausefinallytheyhaveanametoAmajorareaofresearchinvolves
8、rarecancers.Solidtumorsgivethesuffering.Withnewoptimismintheirvoicestheyoftheliverandthyroid,cancerofthebloodandmelanoma,oftenask:“
