https://www.statnews.com/2025/08/21/sarepta-gene-therapy-families-left-behind/
Rare disease patients left adrift after Sarepta gene therapy exit | STAT
Oct 10, 2025 - After deaths and FDA setbacks, Sarepta abandoned LGMD gene therapy, leaving families desperate and patients without long-promised treatments.
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https://www.cnbc.com/2026/01/16/keeping-us-biotech-ahead-of-china-could-aid-rare-disease-patients-experts.html
Keeping U.S. biotech ahead of China could aid rare disease patients: Experts
Jan 16, 2026 - China is becoming a leader in biotech innovation. That offers hope to rare disease patients and presents a problem to American companies trying to save them.
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https://www.mmitnetwork.com/use-case-download-norstellalinq-uncovering-new-rare-disease-patients-using-structured-unstructured-real-world-data/
Use Case Download: NorstellaLinQ: Uncovering New Rare Disease Patients Using Structured and...
Aug 19, 2025 - Discover how MMIT’s NorstellaLinQ combines structured and unstructured real-world data to quantify rare disease patients and support payer and physician...
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https://www.genezen.com/insights/serving-patients-a-shared-mission-in-rare-disease-gene-therapy/
Genezen | Serving Patients Through Rare Disease Gene Therapy Innovation
Nov 17, 2025 - At Genezen, our mission has always been rooted in a singular goal—serving patients. That ethos came to life during a recent panel discussion led by...
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https://jdcorporateblog.com/jd-health-expands-rare-disease-support-assists-over-23000-patients-via-joint-charity-programs/
JD Health Expands Rare Disease Support, Assists Over 23,000 Patients via Joint Charity Programs -...
In observance of Rare Disease Day on February 28, JD Health hosted the 4th Rare Disease Service Ecosystem Forum in Beijing. At the event, JD Health, in...
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