home.social

#rarediseases — Public Fediverse posts

Live and recent posts from across the Fediverse tagged #rarediseases, aggregated by home.social.

fetched live
  1. Sanofi (ENXTPA:SAN) On TD Cowen Spotlight And The Case For A Higher Valuation

    Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply…
    #France #FR #Europe #EU #Sanofi #Cowen #fairvalue #immunology #individualinvestors #rarediseases
    europesays.com/france/67000/

  2. Sanofi (ENXTPA:SAN) On TD Cowen Spotlight And The Case For A Higher Valuation

    Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply…
    #France #FR #Europe #EU #Sanofi #Cowen #fairvalue #immunology #individualinvestors #rarediseases
    europesays.com/france/66974/

  3. Tavneos trial flagged for ‘serious breaches’ of protocol as EU regulators dissect market withdrawal decision

    With the European Union formally reversing its marketing authorization for rare disease med Tavneos last week, regulators from…
    #Europe #EU #Amgen #Autoimmunediseases #ChemoCentryx #CHMPrecommendation #clinicaldata #CSL #drugsafety #EuropeanCommission #EuropeanMedicinesAgency #EuropeanUnion #FiercePharmaHomepage #Pharma #rarediseases #TAVNEOS
    europesays.com/europe/116043/

  4. Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

    https://rbfirehose.com/2026/08/13/shane-the-gamer-belgian-gamer-michiel-vandeweert-dies-at-28-doubling-progeria-life-expectancy/
  5. Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

    https://rbfirehose.com/2026/08/13/shane-the-gamer-belgian-gamer-michiel-vandeweert-dies-at-28-doubling-progeria-life-expectancy/
  6. Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

    https://rbfirehose.com/2026/08/13/shane-the-gamer-belgian-gamer-michiel-vandeweert-dies-at-28-doubling-progeria-life-expectancy/
  7. Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

    https://rbfirehose.com/2026/08/13/shane-the-gamer-belgian-gamer-michiel-vandeweert-dies-at-28-doubling-progeria-life-expectancy/
  8. Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

    https://rbfirehose.com/2026/08/13/shane-the-gamer-belgian-gamer-michiel-vandeweert-dies-at-28-doubling-progeria-life-expectancy/
  9. Sanofi experiencing shortage of two Pompe disease drugs

    Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He…
    #France #FR #Europe #EU #Sanofi #biotechnology #Pharmaceuticals #rarediseases #STAT+
    europesays.com/france/63252/

  10. This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
    First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
    👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
    📱 An app to reliably track a patients' symptoms as a diagnostic aide
    👉 link.europa.eu/GYhXKX
    #IHITransformingHealth #HorizonEU #HorizonEurope

  11. This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
    First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
    👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
    📱 An app to reliably track a patients' symptoms as a diagnostic aide
    👉 link.europa.eu/GYhXKX
    #IHITransformingHealth #HorizonEU #HorizonEurope

  12. This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
    First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
    👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
    📱 An app to reliably track a patients' symptoms as a diagnostic aide
    👉 link.europa.eu/GYhXKX
    #IHITransformingHealth #HorizonEU #HorizonEurope

  13. This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
    First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
    👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
    📱 An app to reliably track a patients' symptoms as a diagnostic aide
    👉 link.europa.eu/GYhXKX
    #IHITransformingHealth #HorizonEU #HorizonEurope

  14. This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
    First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
    👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
    📱 An app to reliably track a patients' symptoms as a diagnostic aide
    👉 link.europa.eu/GYhXKX
    #IHITransformingHealth #HorizonEU #HorizonEurope

  15. Sobi’s dealmaker CEO exits after global expansion

    Why it matters: The departure provides a clear moment to assess one of the more consequential chief executive…
    #Europe #EU #European #rarediseases
    europesays.com/europe/108428/

  16. Rare disease drugmakers seek exclusion from Medicare pricing plans

    WASHINGTON — Biotech companies are lobbying the Trump administration to exclude treatments for rare diseases from programs that…
    #NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Healthcare #biotechnology #CMS #drugdevelopment #drugprices #Health #Pharmaceuticals #Policy #rarediseases #Research #STAT+ #WhiteHouse
    newsbeep.com/us/775982/

  17. Rare disease drugmakers seek exclusion from Medicare pricing plans

    WASHINGTON — Biotech companies are lobbying the Trump administration to exclude treatments for rare diseases from programs that…
    #NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Healthcare #biotechnology #CMS #drugdevelopment #drugprices #Health #Pharmaceuticals #Policy #rarediseases #Research #STAT+ #WhiteHouse
    newsbeep.com/us/775982/

  18. Polio survivor who was last American iron lung user dies at age 78

    Dr Jessica Gray reveals why Americans are living longer Family medicine physician Dr. Jessica Gray explains that new…
    #NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Health #healthcarehouseofrepresentativespolitics #Lifestyle #rarediseases #Vaccines
    newsbeep.com/us/758268/

  19. ARPA-H invests $160 million to make custom gene editing routine

    ARPA-H, the U.S.’ “moonshot” agency for health research, announced Thursday that it will spend up to $160 million…
    #NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Healthcare #biotechnology #crispr #Geneediting #GeneTherapy #Health #hhs #rarediseases #STAT+
    newsbeep.com/us/753035/

  20. ARPA-H invests $160 million to make custom gene editing routine

    ARPA-H, the U.S.’ “moonshot” agency for health research, announced Thursday that it will spend up to $160 million…
    #NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Healthcare #biotechnology #crispr #Geneediting #GeneTherapy #Health #hhs #rarediseases #STAT+
    newsbeep.com/us/753035/

  21. Novartis receives European Commission approval for Itvisma® for spinal muscular atrophy (SMA)

    First gene replacement therapy in the EU for broad population with SMA, including children two years and older,…
    #Europe #EU #EuropeanCommission #CellandGeneTherapy #neuroscience #Novartis #rarediseases #ReimagineMedicine
    europesays.com/europe/84540/

  22. Calico targets longevity pathway with rare disease win

    FDA Breakthrough Therapy Designation for fosigotifator highlights a mechanism that extends well beyond an ultra-rare childhood disorder. Calico…
    #NewsBeep #News #Healthcare #AU #Australia #calico #drugdevelopment #FDA #FDAbreakthroughstatus #fosigotifator #Health #ISRIB #leukodystrophy #RareDiseases #VanishingWhiteMatterdisease
    newsbeep.com/au/770666/

  23. Sanofi (ENXTPA:SAN) Faces EU Antitrust Probe As Nexviazyme Trial Delivers Positive Data

    Find your next quality investment with Simply Wall St’s easy and powerful screener, trusted by over 7 million…
    #France #FR #Europe #EU #Sanofi #antitrustinvestigation #EuropeanCommission #EuropeanEconomicArea #fluvaccine #Pompedisease #rarediseases #SimplyWallSt
    europesays.com/france/43941/

  24. Sanofi (ENXTPA:SAN) Faces EU Antitrust Probe As Nexviazyme Trial Delivers Positive Data

    Find your next quality investment with Simply Wall St’s easy and powerful screener, trusted by over 7 million…
    #Europe #EU #antitrustinvestigation #EuropeanCommission #europeaneconomicarea #EuropeanUnion #fluvaccine #Pompedisease #rarediseases #Sanofi #SimplyWallSt
    europesays.com/europe/82995/

  25. Dangerous tick-borne virus spreading to more Americans, experts warn

    NEWYou can now listen to Fox News articles! A rare and potentially fatal tick-borne illness currently spreading across…
    #NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Health #infectiousdisease #Insects #Lifestyle #outbreaks #outdoorlivingrealestatelifestyle #rarediseases #Viruses
    newsbeep.com/us/733680/

  26. Elixirgen Therapeutics and Nippon Shinyaku Enter into an Option Agreement for the Development and Commercialization of EXG-7001 for Duchenne Muscular Dystrophy

    Elixirgen Therapeutics, Inc. BALTIMORE, June 26, 2026 (GLOBE NEWSWIRE) — Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company developing…
    #EuropeSays #Japan #JP #biotechnologycompany #Nippon #NipponShinyaku #rarediseases #Therapeutics
    europesays.com/japan/49112/

  27. Elixirgen Therapeutics and Nippon Shinyaku Enter into an Option Agreement for the Development and Commercialization of EXG-7001 for Duchenne Muscular Dystrophy

    Elixirgen Therapeutics, Inc. BALTIMORE, June 26, 2026 (GLOBE NEWSWIRE) — Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company developing…
    #EuropeSays #Japan #JP #biotechnologycompany #Nippon #NipponShinyaku #rarediseases #Therapeutics
    europesays.com/japan/47924/

  28. Press Release: Sanofi appoints Paulo Fontoura as Global Head of R&D

    Sanofi Winthrop Industrie Sanofi appoints Paulo Fontoura as Global Head of R&D Paris, June 22, 2026. Sanofi today announced the appointment of Paulo …
    #France #FR #Europe #EU #Sanofi #ChiefExecutiveOfficer #ChiefMedicalOfficer #clinicaldevelopment #ExecutiveCommittee #ExecutiveVicePresident #Fontoura #GlobalHeadofResearch&DevelopmentPharma #HoumanAshrafian #rarediseases #therapeuticareas
    europesays.com/france/40192/

  29. Press Release: Sanofi appoints Paulo Fontoura as Global Head of R&D

    Sanofi Winthrop Industrie Sanofi appoints Paulo Fontoura as Global Head of R&D Paris, June 22, 2026. Sanofi today announced the appointment of Paulo …
    #France #FR #Europe #EU #Sanofi #ChiefExecutiveOfficer #ChiefMedicalOfficer #clinicaldevelopment #ExecutiveCommittee #ExecutiveVicePresident #Fontoura #GlobalHeadofResearch&DevelopmentPharma #HoumanAshrafian #rarediseases #therapeuticareas
    europesays.com/france/39248/

  30. 𝗥𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝘁𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗱𝗲𝗺𝗮𝗻𝗱 𝗮𝗰𝗿𝗼𝘀𝘀 𝘁𝗵𝗲 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗶𝘀 𝗲𝗻𝘁𝗲𝗿𝗶𝗻𝗴 𝗮 𝗵𝗶𝗴𝗵 𝗴𝗿𝗼𝘄𝘁𝗵 𝗵𝗲𝗮𝗹𝘁𝗵𝗰𝗮𝗿𝗲 𝗽𝗵𝗮𝘀𝗲!
    The 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗠𝗮𝗿𝗸𝗲𝘁 is expanding as governments improve healthcare access, diagnostics, and specialty drug availability. Rising awareness and advancements in personalized medicine are reshaping patient care outcomes.
    𝗥𝗲𝗮𝗱 𝗺𝗼𝗿𝗲: kenresearch.com/middle-east-ra
    #Healthcare #RareDiseases #MiddleEast #PharmaIndustry

  31. 𝗥𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝘁𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗱𝗲𝗺𝗮𝗻𝗱 𝗮𝗰𝗿𝗼𝘀𝘀 𝘁𝗵𝗲 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗶𝘀 𝗲𝗻𝘁𝗲𝗿𝗶𝗻𝗴 𝗮 𝗵𝗶𝗴𝗵 𝗴𝗿𝗼𝘄𝘁𝗵 𝗵𝗲𝗮𝗹𝘁𝗵𝗰𝗮𝗿𝗲 𝗽𝗵𝗮𝘀𝗲!
    The 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗠𝗮𝗿𝗸𝗲𝘁 is expanding as governments improve healthcare access, diagnostics, and specialty drug availability. Rising awareness and advancements in personalized medicine are reshaping patient care outcomes.
    𝗥𝗲𝗮𝗱 𝗺𝗼𝗿𝗲: kenresearch.com/middle-east-ra
    #Healthcare #RareDiseases #MiddleEast #PharmaIndustry

  32. 𝗥𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝘁𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗱𝗲𝗺𝗮𝗻𝗱 𝗮𝗰𝗿𝗼𝘀𝘀 𝘁𝗵𝗲 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗶𝘀 𝗲𝗻𝘁𝗲𝗿𝗶𝗻𝗴 𝗮 𝗵𝗶𝗴𝗵 𝗴𝗿𝗼𝘄𝘁𝗵 𝗵𝗲𝗮𝗹𝘁𝗵𝗰𝗮𝗿𝗲 𝗽𝗵𝗮𝘀𝗲!
    The 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗠𝗮𝗿𝗸𝗲𝘁 is expanding as governments improve healthcare access, diagnostics, and specialty drug availability. Rising awareness and advancements in personalized medicine are reshaping patient care outcomes.
    𝗥𝗲𝗮𝗱 𝗺𝗼𝗿𝗲: kenresearch.com/middle-east-ra
    #Healthcare #RareDiseases #MiddleEast #PharmaIndustry

  33. Soligenix advances rare disease innovation using platform science to expand its therapeutic pipeline and address unmet medical needs affecting millions. #RareDiseases #Biotech

  34. Soligenix advances rare disease innovation using platform science to expand its therapeutic pipeline and address unmet medical needs affecting millions. #RareDiseases #Biotech

  35. Soligenix advances rare disease innovation using platform science to expand its therapeutic pipeline and address unmet medical needs affecting millions. #RareDiseases #Biotech

  36. Soligenix advances rare disease innovation using platform science to expand its therapeutic pipeline and address unmet medical needs affecting millions. #RareDiseases #Biotech

  37. Soligenix reports strong Phase 3 HyBryte data for cutaneous T-cell lymphoma, with response rates exceeding expectations. Interim analysis could accelerate commercialization path. #Biotech #RareDiseases

  38. Soligenix reports strong Phase 3 HyBryte data for cutaneous T-cell lymphoma, with response rates exceeding expectations. Interim analysis could accelerate commercialization path. #Biotech #RareDiseases

  39. Soligenix reports strong Phase 3 HyBryte data for cutaneous T-cell lymphoma, with response rates exceeding expectations. Interim analysis could accelerate commercialization path. #Biotech #RareDiseases

  40. Soligenix reports strong Phase 3 HyBryte data for cutaneous T-cell lymphoma, with response rates exceeding expectations. Interim analysis could accelerate commercialization path. #Biotech #RareDiseases

  41. Soligenix publishes peer-reviewed HyBryte clinical data demonstrating safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 trial interim results expected Q2 2026. #Biotech #RareDiseases