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Why patient-centricity is key for rare disease clinical research

Adopting patient-centric approaches to help overcome the unique challenges related to clinical trials for rare diseases.

United statesCezary statuchAlexion pharmaceuticalsTranslational sciencesHead of medical affairs internationalHead of medical affairsEuropean commissionMember cancer centersOffice of rare diseases researchNational centerChina medical affairs networkPublic healthCommittee of the medical affairs professional societyClinical trials dayMedical affairs internationalLatin america

FDA Approves B-VEC Topical Gene Therapy for Dystrophic Epidermolysis Bullosa

The long-awaited FDA approval follows data that demonstrated about 70% wound closure by 6 months in dystrophic epidermolysis bullosa patients treated with B-VEC.

United statesBeremagene geperpavec svdtM peter marinkovichStanford university school of medicineDisease clinic at stanford health careKrystal biotech incTranslational sciencesAmerican academy of dermatologyNational centerNational institutes of healthOffice of rare diseases researchDrug administrationAdvancing translationalBlistering disease clinicStanford health careAssociate professor

Butterfly Disease: A Visible Condition in Need of a Cure

Patients with dystrophic epidermolysis bullosa face near-constant pain and discomfort. While there are several measures clinicians have to help them, a curative treatment remains elusive.

Karan lalPeter marinkovichUs national library of medicineNational institutes of healthTranslational sciencesOffice of rare diseases researchNational centerStanford university school of medicineDrug administrationAdvancing translationalAffiliated dermatology scottsdaleStanford university schoolDystrophic epidermolysisAccessed marchEpidermolysis bullosa frequently askedStanford medicine dermatology

RARE Drug Development Symposium: Fostering Successful Connections Between Stakeholders to Accelerate Progress

RARE Drug Development Symposium: Fostering Successful Connections Between Stakeholders to Accelerate Progress
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United statesYssa dewoodyAdeline vanderverIlene penn millerIlene millerRare disease clinical research networkAmerican epilepsy societyEpilepsy leadership council advocacy coCommon fund undiagnosed disease networkRare epilepsy network ex officioOffice of rare diseases researchEpilepsy research benchmarksFoundation cooperative agreementInternational cofounderCommission for neurodevelopmental cnvsData management coordinating center

BioSpace Movers & Shakers, March 4

Biopharma and life sciences companies strengthen their leadership teams and boards with these Movers & Shakers.

United statesFrance generalBerlin centerUniversity of pennsylvaniaSouth koreaSouth koreanJohann bauersachsTechnische universitAdamb hughesLiodmila tchistiakovaGerasimos filippatosEric durandJodi parramonAlethia youngJohn farquharBirgitte stephenson