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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

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

Rare Disease Day at NIH 2024 | National Center for Advancing Translational Sciences

Rare Disease Day at NIH 2024 | National Center for Advancing Translational Sciences
nih.gov - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from nih.gov Daily Mail and Mail on Sunday newspapers.

United statesNational institute on alcohol abuseClinical centerMain campus natcher conference center onBlood instituteRare diseases researchDivision of rare diseases research innovationNational institute of neurological disordersNational heartDrug administrationNational cancer instituteEverylife foundation for rare diseasesDiseases network participant engagementNational organization for rare disordersRare disease dayDisease day

Kelly: Patients with rare diseases deserve comparable treatment

Kelly: Patients with rare diseases deserve comparable treatment
riponadvance.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from riponadvance.com Daily Mail and Mail on Sunday newspapers.

Mike kellyBarth syndrome foundationEverylife foundation for rare diseasesAutoinflammatory allianceUnited mitochondrial disease foundationRare disease diversity councilNational organization for rare disordersUnited porphyrias associationProviding realistic opportunity to equalComparable treatmentRare actDoris matsuiHaystack projectEverylife foundationRare diseasesNational organization