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The immune system can sabotage gene therapies — can scientists rein it in?

People treated with gene therapy cannot receive a second dose for fear of a dangerous immune response. Researchers hope to find a way around this. People treated with gene therapy cannot receive a second dose for fear of a dangerous immune response. Researchers hope to find a way around this.

United statesNew yorkUniversity of washingtonUnited kingdomUniversity of pennsylvaniaJesse gelsingerLindsey georgeNicholas giovannoneJannie kennedyMartin kangChris wrightJulie crudeleUniversity of south carolinaEverylife foundation for rare diseasesAmerican society of geneScience photo library

Avidity Biosciences Honors Rare Disease Day®

SAN DIEGO, Feb. 29, 2024 /PRNewswire/ Avidity Biosciences, Inc. , a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates ,.

San diegoUnited statesSarah boyceNavjot raiGeoffrey grandeJannie kennedyPrnewswire avidity biosciences incExchange commissionAvidity biosciences incAntibody oligonucleotide conjugatesRare diseaseRare disease dayPatient engagementEverylife foundationDisease dayMyotonic dystrophy type

Limb-Girdle Muscular Dystrophy Leaders Advance Collaborative Dialogue to Accelerate Drug Development to Address the Urgent Unmet Need in Limb-Girdle Muscular Dystrophy

/PRNewswire/ On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), convened a.

United statesDouglas sprouleMichelle campbellMatthew wicklundJannie kennedyKatherine mathewsKathryn bryant knudsonNicholas johnsonPeter marksLouise rodino klapacNicole verdunLindsay alfanoPeter kangPeter steinJames signorovitchJerryr mendell

Empowering : Rare Disease Week in DC to draw calls for change – NBC4 Washington

Patients will gather on Capitol Hill to talk to members of Congress and build community as part of Rare Disease Week.

United statesVirginia beachNell choiMarie woodLauren stanfordStephanie riordanNational organization for rare disordersChildren nationalRare pediatric disease priority review voucher programEverylife foundation for rare diseasesDrug administrationChildren health insurance programCapitol hillRare diseaseRare disease legislative advocatesEverylife foundation

Dillingham fifth-grader wins national art prize

Dillingham fifth-grader wins national art prize
kdlg.org - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from kdlg.org Daily Mail and Mail on Sunday newspapers.

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