#rarediseases — Public Fediverse posts

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
https://www.newsbeep.com/us/775982/

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
https://www.newsbeep.com/us/775982/

https://www.europesays.com/ie/578567/ MEP calls for Ireland to increase testing done on newborn babies for rare conditions – The Journal #BillyKelleher #Éire #Entertainment #EU #EuropeanParliament #HeelPrickTest #IE #Ireland #RareDiseases

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+
https://www.newsbeep.com/us/753035/

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+
https://www.newsbeep.com/us/753035/

How one father built a biotech, Grace Science, to save his daughter

Highlights Matt Wilsey has spent a decade and $70 million trying to develop a cure for his daughter…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Healthcare #biotechnology #drugdevelopment #FDA #GeneTherapy #Health #Pharmaceuticals #rarediseases #STAT+
https://www.newsbeep.com/us/752485/

How one father built a biotech, Grace Science, to save his daughter

Highlights Matt Wilsey has spent a decade and $70 million trying to develop a cure for his daughter…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Healthcare #biotechnology #drugdevelopment #FDA #GeneTherapy #Health #Pharmaceuticals #rarediseases #STAT+
https://www.newsbeep.com/us/752485/

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
https://www.europesays.com/europe/84540/

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
https://www.europesays.com/france/43941/

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
https://www.europesays.com/europe/82995/

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
https://www.newsbeep.com/us/733680/

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
https://www.newsbeep.com/us/733680/

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
https://www.europesays.com/japan/49112/

Incyte (INCY) Wins EU Backing For Opzelura As Pipeline Data Builds Across Four Drugs

Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply…
#Europe #EU #atopicdermatitis #EuropeanCommission #EuropeanUnion #globalcommunity #Incyte #individualinvestors #myeloproliferativeneoplasms #Opzelura #rareconditions #rarediseases #regulatoryupdates #treatmentguidelines
https://www.europesays.com/europe/80625/

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
https://www.europesays.com/japan/47924/

https://www.europesays.com/ch/86554/ Enspryng falls short of Roche’s expectations in myasthenia gravis #AutoimmuneDiseases #Chugai #ENSPRYNG #FiercePharmaHomepage #MyastheniaGravis #Pharma #RareDiseases #Roche

https://www.europesays.com/ch/86454/ Roche’s Enspryng cuts MOGAD relapse risk by 68% in phase 3 #AmericanAcademyOfNeurology #AutoimmuneDiseases #ClinicalData #ENSPRYNG #FiercePharmaHomepage #Pharma #RareDiseases #Roche

https://www.europesays.com/dk/107515/ Sobi to present new clinical and real-world data at EHA 2026 in Stockholm #EuropeanUnion #HaemophagocyticLymphohistiocytosis #ImmuneThrombocytopenia #myelofibrosis #ParoxysmalNocturnalHaemoglobinuria #pegcetacoplan #PosterPresentations #RareDiseases #RealWorldData #Sobi #Stockholm #Sweden

https://www.europesays.com/ie/533312/ Long-Read DNA Test Could Replace 15 Existing Tests for Rare Diseases #diagnosis #DnaSequencing(sequencing) #Éire #Health #IE #Ireland #LongReadSequencing #MolecularDx #News&Features #RareDiseases

https://www.europesays.com/ch/82249/ Novartis IgAN data in The Lancet show clinically meaningful slowing of kidney function decline with Vanrafia® over 2.5 years #ClinicalTrials #Innovation #Novartis #RareDiseases #ReimagineMedicine

https://www.europesays.com/ch/82235/ Novartis dystrophy data tee up regulatory talks on Avidity drug #biotech #ClinicalData #FierceBiotechHomepage #MuscularDystrophy #Novartis #RareDiseases #siRNA

https://www.europesays.com/ch/81789/ Hansoh, Chugai ink separate and lucrative licensing agreements #AntibodyDrugConjugates #Asia #biologics #biotech #china #ChugaiPharmaceutical #ColorectalCancer #Deals #DrugDelivery #FierceBiotechHomepage #HansohPharma #JAPAN #LicensingDeals #Milestones #RaniTherapeutics #RareDiseases #Roche #royalties #UnitedStates

https://www.europesays.com/ch/77782/ Novartis IgAN data in The Lancet show clinically meaningful #ClinicalTrials #Innovation #MediaRelease #Novartis #NovartisPharmaAG #RareDiseases #ReimagineMedicine #SwissStockExchange:NOVNSW

AstraZeneca Reports Robust Q1 Growth and Advances High-Value Drug Pipeline

astrazenica ©Shutterstock AstraZeneca plc (LSE:AZN) delivered a strong start to 2026, posting first-quarter total revenue of $15.3 billion,…
#EuropeSays #Britain #Europe #EU #AstraZeneca #AstraZenecaPLC #earningspershare #oncologytreatments #rarediseases #Strategicpartnerships
https://www.europesays.com/britain/55463/

https://www.europesays.com/britain/55463/ AstraZeneca Reports Robust Q1 Growth and Advances High-Value Drug Pipeline #AstraZeneca #AstraZenecaPLC #EarningsPerShare #OncologyTreatments #RareDiseases #StrategicPartnerships

#VeteransAdministration #RareDiseases #Military

From Stars and Stripes: Veteran with rare muscle-wasting disease seeks VA recognition

https://www.stripes.com/veterans/2026-05-29/veteran-rare-disease-va-recognition-21816111.html

#VeteransAdministration #RareDiseases #Military

From Stars and Stripes: Veteran with rare muscle-wasting disease seeks VA recognition

https://www.stripes.com/veterans/2026-05-29/veteran-rare-disease-va-recognition-21816111.html

#VeteransAdministration #RareDiseases #Military

From Stars and Stripes: Veteran with rare muscle-wasting disease seeks VA recognition

https://www.stripes.com/veterans/2026-05-29/veteran-rare-disease-va-recognition-21816111.html

https://www.europesays.com/ch/74042/ New Novartis data at ERA 2026 advance scientific #ClinicalScience #ClinicalTrials #MediaRelease #Novartis #NovartisPharmaAG #RareDiseases #ReimagineMedicine #SwissStockExchange:NOVNSW

Sanofi unveils AI-powered platform for early rare disease detection | Company News

The free web-based tool enables healthcare professionals to input a patient’s symptoms, medical history and test…
#France #FR #Europe #EU #Sanofi #AccelRare #AIdiagnosistool #CentresofExcellence #healthcareai #medicaldiagnostics #MedVir #rarediseasedetection #rarediseases #SanofiIndia
https://www.europesays.com/france/25730/

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: https://www.kenresearch.com/middle-east-rare-diseases-treatment-market
#Healthcare #RareDiseases #MiddleEast #PharmaIndustry

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

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

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

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

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

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

Soligenix publishes HyBryte clinical data in peer-reviewed journal, highlighting safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 interim analysis expected Q2 2026. #Biotech #RareDiseases

Soligenix publishes HyBryte clinical data in peer-reviewed journal, highlighting safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 interim analysis expected Q2 2026. #Biotech #RareDiseases

Soligenix's SGX945 receives Promising Innovative Medicine designation from UK MHRA for Behçet's disease, advancing rare disease treatment development. #Biotech #RareDiseases

Soligenix's SGX945 receives Promising Innovative Medicine designation from UK MHRA for Behçet's disease, advancing rare disease treatment development. #Biotech #RareDiseases

Soligenix approaches pivotal 2026 with Phase 3 HyBryte trial for CTCL and SGX945 advancement for Behçet's disease. Multiple clinical catalysts expected. #Biotech #RareDiseases

Soligenix approaches pivotal 2026 with Phase 3 HyBryte trial for CTCL and SGX945 advancement for Behçet's disease. Multiple clinical catalysts expected. #Biotech #RareDiseases

Soligenix enters a pivotal 2026 with key clinical milestones ahead, including Phase 3 FLASH2 trial data for HyBryte in CTCL treatment and progress on SGX945 for Behçet's disease. #Biotech #RareDiseases

Soligenix enters a pivotal 2026 with key clinical milestones ahead, including Phase 3 FLASH2 trial data for HyBryte in CTCL treatment and progress on SGX945 for Behçet's disease. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behçet Disease, securing 10 years of market exclusivity. Phase 2a data demonstrated efficacy and safety. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behçet Disease, securing 10 years of market exclusivity. Phase 2a data demonstrated efficacy and safety. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behcet Disease, securing 10 years of market exclusivity. Builds on Phase 2a efficacy data. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behcet Disease, securing 10 years of market exclusivity. Builds on Phase 2a efficacy data. #Biotech #RareDiseases

Soligenix advances Phase 3 FLASH2 trial for cutaneous T-cell lymphoma with interim analysis expected Q2 2026. Company also secured orphan drug designation for dusquetide in Behçet's Disease. #Biotech #RareDiseases