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Local family fundraising to support gene therapy research for baby’s rare condition - FlamboroughToday.comFlamboroughToday.com · Sat, 08 Aug 2026 14:16:10 GMTCongressional Panel Says the United States Is Losing Its Lead on Rare Disease Cures - Medical DailyMedical Daily · Sat, 08 Aug 2026 03:30:41 GMTExeter family rallies community with 5K after daughters’ rare‑disease diagnosis - Seacoastonline.comSeacoastonline.com · Fri, 07 Aug 2026 18:51:00 GMTEnsoma sheds more staff, pauses preclinical programs to home in on lead gene therapy - Fierce BiotechFierce Biotech · Fri, 07 Aug 2026 15:16:34 GMTBioMarin axes $270M asset after missing phase 3 endpoint - Fierce BiotechFierce Biotech · Fri, 07 Aug 2026 13:10:41 GMTStudy identifies promising therapy to prevent relapses in rare neurological disease - Medical XpressMedical Xpress · Fri, 07 Aug 2026 12:00:03 GMTBoosted by new rare disease pillar, Merck KGaA lifts sales outlook for the year - FirstWord PHARMAFirstWord PHARMA · Thu, 06 Aug 2026 19:16:57 GMTCAMP4 advances rare disease treatment for SYNGAP1 into human trials - CNBCCNBC · Thu, 06 Aug 2026 18:52:12 GMT