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Rare Disease · news · pharmadog

← back to Rare Disease

News · Rare Disease

856 stories

Jul 16, 26Chutes & Ladders—Cue lines up new CMO and research headfiercebiotech

Jul 16, 26STAT+: Amylyx nears pivotal endocrine drug study readoutstat

Jul 16, 26STAT+: CMS signals intent to revamp how it pays for clinical software and AIstat

Jul 15, 26Graham’s death from aortic dissection raises questions about preventing the rare disorderstat

Jul 15, 26Graham’s death from aortic dissection raises questions about preventing the rare disorderstat

Jul 15, 26STAT+: New study untangles how Epstein-Barr viral infection triggers immune response in multiple sclerosisstat

Jul 15, 26STAT+: Celcuity’s breast cancer drug approved, its firststat

Jul 15, 26More health workers strike as Ebola cases in Congo exceed 2,000, including 754 deathsstat

Jul 15, 26Why Axiom hopes it’s ‘solved 2 major potholes’ that have hurt other stem cell biotechsfiercebiotech

Jul 15, 26STAT+: A new biotech startup tries to tackle obesity, but not in the way you might expectstat

Jul 14, 26Majority of new Ebola cases in eastern Congo are from unknown chains, as outbreak outpaces responsestat

Jul 13, 26Building a workforce engine for biomanufacturingfiercebiotech

Jul 13, 26Opinion: Is there a doctor on board? Yes, and airlines depend on itstat

Jul 10, 26STAT+: Roche ends Huntington’s gene-silencing programsstat

Jul 10, 26STAT+: Pharmalittle: We’re reading about bigger drug discounts in Germany, drugmakers embracing secrecy, and morestat

Jul 10, 26ARPA-H puts $160M into bespoke drug therapies; Biohaven switches up scientific leadershipbiopharma dive

Jul 10, 26FDA quietly pushes back deadline on electric shock banstat

Jul 10, 26STAT+: How a Boston doctor built a following as a ‘loud and unafraid’ voice in the Trump erastat

Jul 9, 26Roche scraps two Ionis-partnered Huntington’s drugsbiopharma dive

Jul 9, 26ARPA-H bookmarks $160M for custom gene editing treatments. Is it enough?endpoints

Jul 9, 26STAT+: ARPA-H launches $160 million effort to develop custom gene editing drugsstat

Jul 9, 26STAT+: Prime wins Beam arbitration, clearing path to clinicstat

Jul 9, 26STAT+: Pharmalittle: We’re reading about FDA commissioner contenders, a heart drug failure, and morestat

Jul 9, 26Who’s going to run the FDA?stat

Jul 9, 26STAT+: AstraZeneca, Ionis report major trial failure with heart disease drugstat

Jul 9, 26STAT+: The quest to save Grace — and clear the way for rare disease patients everywherestat

Jul 9, 26Early Alert: Convenience Kits Issue from Medlinefda medwatch

Jul 8, 26Transplants extended survival for patients whose stage 4 lung cancer hadn’t spread, study saysstat

Jul 8, 26STAT+: Prime Medicine claims victory in dispute over gene-editing technology with Beam Therapeuticsstat

Jul 8, 26STAT+: Pharmalittle: We’re reading about a Vera kidney drug approval, a U.K. Enhertu pricing deal, and morestat

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