Abstract
Get full access to this article
View all access options for this article.
References
1.Sarepta Therapeutics . Sarepta Therapeutics Announces Voluntary Pause of ELEVIDYS Shipments in the U.S . 2025 . Available from: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys [Last accessed:
August 1 , 2025
].
2.U.S. Food and Drug Administration . FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients . 2025 . Available from: https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients [Last accessed:
August 1 , 2025
].
3.
Smith
G
,
Zhang
RC
,
Tong
A
. FDA’s Prasad Exits Following Political Backlash Over Sarepta . 2025 . Available from: https://www.bloomberg.com/news/articles/2025-07-30/fda-s-vinay-prasad-exits-after-sarepta-gene-therapy-controversy [Last accessed:
August 1 , 2025
].
4.Rocket Pharmaceuticals . Rocket Pharmaceuticals Announces Strategic Corporate Reorganization and Pipeline Prioritization of Cardiovascular Programs . 2025 . Available from: https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-strategic-corporate [Last accessed:
July 26 , 2025
].
5.Rocket Pharmaceuticals . Form 8-K . 2025 . Available from: https://rocketpharmaceuticals.gcs-web.com/static-files/2012f8e6-920e-4bbb-9a4e-a8fc195cd820 [Last accessed:
July 26 , 2025
].
6.Ultragenyx Pharmaceuticals . Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) . 2025 . Available from: https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-receives-complete-response-letter-fda-ux111-aav-gene [Last accessed:
July 19 , 2025
].
7.Sangamo Therapeutics . Sangamo Therapeutics Announces Positive Topline Results From Registrational STAAR Study in Fabry Disease . 2025 . Available from: https://investor.sangamo.com/news-releases/news-release-details/sangamo-therapeutics-announces-positive-topline-results [Last accessed:
July 25 , 2025
].
8.U.S. Centers for Medicare & Medicaid Services . CMS Expands Access to Lifesaving Gene Therapies Through Innovative State Agreements . 2025 . Available from: https://www.hhs.gov/press-room/cms-announces-participation-in-cell-and-gene-therapy-access-model.html [Last accessed:
August 1 , 2025
].
9.Genascence . Genascence Announces U.S. Food and Drug Administration Grants Regenerative Medicine Advanced Therapy Designation to GNSC-001 for Knee Osteoarthritis (OA) . 2025 . Available from: https://www.businesswire.com/news/home/20250716308556/en/Genascence-Announces-U.S.-Food-and-Drug-Administration-Grants-Regenerative-Medicine-Advanced-Therapy-Designation-to-GNSC-001-for-Knee-Osteoarthritis-OA [Last accessed:
July 19 , 2025
].