#orphandrug — Public Fediverse posts
Live and recent posts from across the Fediverse tagged #orphandrug, aggregated by home.social.
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AstraZeneca updates Ultomiris HSCT-TMA Phase III data | AZN SEC Filing
FORM 6-K SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Report of Foreign Issuer Pursuant…
#EuropeSays #Britain #Europe #EU #AstraZeneca #AZN #BreakthroughTherapy #clinicaldata #HSCT-TMA #OrphanDrug #paediatricsurvival #PhaseIIItrial #SECfiling #Ultomiris
https://www.europesays.com/britain/91592/ -
https://www.europesays.com/britain/91592/ AstraZeneca updates Ultomiris HSCT-TMA Phase III data | AZN SEC Filing #AstraZeneca #AZN #BreakthroughTherapy #ClinicalData #HSCTTMA #OrphanDrug #PaediatricSurvival #PhaseIIITrial #SECFiling #Ultomiris
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Quoin completes Japan subsidiary for QRX003
Quoin Pharmaceuticals (NASDAQ: QNRX) has completed establishing a wholly-owned subsidiary in Japan to…
#EuropeSays #Japan #JP #commercialization #Japansubsidiary #NethertonSyndrome #orphandrug #QNRX #QRX003 #QuoinPharmaceuticals
https://www.europesays.com/japan/43107/ -
Alkermes Granted Orphan Drug Designations for Alixorexton in US, Europe
Key takeaways: The US FDA granted orphan drug designation to alixorexton for the treatment of idiopathic hypersomnia, while…
#Europe #EU #EuropeanCommission #Alkermes #IdiopathicHypersomnia #Narcolepsy #OrphanDrug #OX2R
https://www.europesays.com/europe/71303/ -
https://www.europesays.com/ch/81579/ Novartis del-brax FSHD trial hits biomarker goals | NVS SEC Filing #(delBrax) #AOCTherapy #AvidityBiosciences #BiomarkerEndpoint #FSHDTrial #NeuromuscularPipeline #Novartis #NVS #OrphanDrug #PhaseIIIStudy
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Oculis Announces European Medicines Agency PRIME Designation for Privosegtor, Advancing a Potential First‑in‑Class Neuroprotective Candidate for Optic Neuritis
Oculis Holding AG ZUG, Switzerland, March 31, 2026 (GLOBE NEWSWIRE) — PRIME designation follows recent Breakthrough Thera…
#Europe #EU #BreakthroughTherapy #European #EuropeanMedicinesAgency #FDA #ischemicopticneuropathy #Oculis #opticneuritis #opticneuropathies #OrphanDrug #Privosegtor #unmetneeds
https://www.europesays.com/europe/1844/ -
https://www.europesays.com/ie/274996/ AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD) #BayerAG #ClinicalTrial #Éire #EnzymeReplacement #FDAFastTrack #FoodAndDrugAdministration #GeneTherapy #Health #HealthCare #Healthcare #IE #InvestigationalNewDrug #Ireland #LOPD #OrphanDrug #PompeDisease
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https://www.europesays.com/uk/311279/ Bridging psychiatry and rare genetic diseases: a scoping review of therapeutic strategies and diagnostic delay paired with healthcare economic burden analysis | Orphanet Journal of Rare Diseases #addiction #BehavioralHealth #DiagnosticDelay #general #Genetics #HumanGenetics #Medication #Medicine/PublicHealth #MentalHealth #OrphanDrug #Pharmacology/Toxicology #psychiatric #RareDisease #Science #TreatmentResistant #UK #UnitedKingdom
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Bridging psychiatry and rare genetic diseases: a scoping review of therapeutic strategies and diagnostic delay paired with healthcare economic burden analysis | Orphanet Journal of Rare Diseases
Nguengang W…
#NewsBeep #News #Genetics #Addiction #Behavioralhealth #Diagnosticdelay #general #HumanGenetics #Medication #Medicine/PublicHealth #Mentalhealth #Orphandrug #Pharmacology/Toxicology #Psychiatric #RareDisease #Science #Treatment-resistant #UK #UnitedKingdom
https://www.newsbeep.com/uk/38985/ -
Bridging psychiatry and rare genetic diseases: a scoping review of therapeutic strategies and diagnostic delay paired with healthcare economic burden analysis | Orphanet Journal of Rare Diseases
Nguengang Wakap…
#NewsBeep #News #Genetics #addiction #AU #Australia #Behavioralhealth #Diagnosticdelay #general #HumanGenetics #Medication #Medicine/PublicHealth #Mentalhealth #Orphandrug #Pharmacology/Toxicology #Psychiatric #raredisease #Science #Treatment-resistant
https://www.newsbeep.com/au/38380/ -
A new company developing treatments for neurological and psychiatric diseases from aberrant immune system activity is raising $58 million in seed funds.
https://sciencebusiness.technewslit.com/?p=45199
#News #Press #Science #Business #Biotechnology #Finance #Autoimmune #Encephalitis #MentalHealth #Psychosis #SeedFunds #VentureCapital #Investment #Preclinical #FDA #OrphanDrug #RareDisease
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A new company developing treatments for neurological and psychiatric diseases from aberrant immune system activity is raising $58 million in seed funds.
https://sciencebusiness.technewslit.com/?p=45199
#News #Press #Science #Business #Biotechnology #Finance #Autoimmune #Encephalitis #MentalHealth #Psychosis #SeedFunds #VentureCapital #Investment #Preclinical #FDA #OrphanDrug #RareDisease
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A new company developing treatments for neurological and psychiatric diseases from aberrant immune system activity is raising $58 million in seed funds.
https://sciencebusiness.technewslit.com/?p=45199
#News #Press #Science #Business #Biotechnology #Finance #Autoimmune #Encephalitis #MentalHealth #Psychosis #SeedFunds #VentureCapital #Investment #Preclinical #FDA #OrphanDrug #RareDisease
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A new company developing treatments for neurological and psychiatric diseases from aberrant immune system activity is raising $58 million in seed funds.
https://sciencebusiness.technewslit.com/?p=45199
#News #Press #Science #Business #Biotechnology #Finance #Autoimmune #Encephalitis #MentalHealth #Psychosis #SeedFunds #VentureCapital #Investment #Preclinical #FDA #OrphanDrug #RareDisease
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#Vutrisiran bei hereditärer Transthyretin-Amyloidose: Anhaltspunkt für geringen Zusatznutzen
Der Hersteller hat die reguläre frühe Nutzenbewertung für sein #OrphanDrug vorgezogen, was seit 2019 möglich ist. Weniger vielversprechende neue Wirkstoffe profitieren weiterhin von der Zusatznutzenfiktion.
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#Vutrisiran bei hereditärer Transthyretin-Amyloidose: Anhaltspunkt für geringen Zusatznutzen
Der Hersteller hat die reguläre frühe Nutzenbewertung für sein #OrphanDrug vorgezogen, was seit 2019 möglich ist. Weniger vielversprechende neue Wirkstoffe profitieren weiterhin von der Zusatznutzenfiktion.
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#Vutrisiran bei hereditärer Transthyretin-Amyloidose: Anhaltspunkt für geringen Zusatznutzen
Der Hersteller hat die reguläre frühe Nutzenbewertung für sein #OrphanDrug vorgezogen, was seit 2019 möglich ist. Weniger vielversprechende neue Wirkstoffe profitieren weiterhin von der Zusatznutzenfiktion.
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#Vutrisiran bei hereditärer Transthyretin-Amyloidose: Anhaltspunkt für geringen Zusatznutzen
Der Hersteller hat die reguläre frühe Nutzenbewertung für sein #OrphanDrug vorgezogen, was seit 2019 möglich ist. Weniger vielversprechende neue Wirkstoffe profitieren weiterhin von der Zusatznutzenfiktion.
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#Vutrisiran bei hereditärer Transthyretin-Amyloidose: Anhaltspunkt für geringen Zusatznutzen
Der Hersteller hat die reguläre frühe Nutzenbewertung für sein #OrphanDrug vorgezogen, was seit 2019 möglich ist. Weniger vielversprechende neue Wirkstoffe profitieren weiterhin von der Zusatznutzenfiktion.