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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    ChemWerth works in generic API development & supply, non-infringement patent strategy development and regulatory support.
    All News #Library
    Rare Diseases

    aTyr Flunks Phase 3 Lung Disease Trial, Deflating Stock

    REUTERS

    Entera Bio Reveal Positive PK Data for Oral GLP-2 Tablet at ESPEN

    GLOBENEWSWIRE

    Assertio Shares SYMPAZAN Oral Film Patient Study Results

    GLOBENEWSWIRE

    Palvella Completes Phase 2 TOIVA Trial Enrollment for QTORIN™

    GLOBENEWSWIRE

    AMO Pharma Reveals REACHCDM-X Study`s Safety Data for AMO-02

    PR NEWSWIRE

    Lion TCR Gets Triple FDA Milestones for Hepatitis B IND Clearance

    PR NEWSWIRE

    SOM Biotech Gets EMA COMP Nod for SOM3355 in Huntington`s Disease

    PHARMAWEB

    Corcept Faces Teva Lawsuit Over Mifepristone `Monopoly`

    REUTERS

    Targeted Myasthenia Gravis Therapies Flood Sparse Market

    BIOSPACE

    Belite Ends 2-Year Ph 3 Tinlarebant Trial in Stargardt Disease

    GLOBENEWSWIRE

    Mandos Health Shows Niemann-Pick C Analyses at 2025 ANA Meet

    BUSINESSWIRE

    David Cameron to Speak at Morgan Stanley Exchange 2025

    PR NEWSWIRE

    Nacuity Pharma Reports Positive NPI-001 Trial for Usher Syndrome

    GLOBENEWSWIRE

    Intercept Withdraws Ocaliva for PBC from US Market, Trial on Hold

    GLOBENEWSWIRE

    Webinar on Why Most Rare Disease Trials Fail, Hosted by Xtalks

    PR NEWSWIRE

    Inpharmus Emerges as MERAT Leader in Rare Diseases, Oncology

    PR NEWSWIRE

    Vertex Win 2025 Lasker~DeBakey Award for Cystic Fibrosis Research

    PHARMAWEB

    Assertio Presents Sympazan Study Data at Neurology Meeting

    GLOBENEWSWIRE

    Avidity` Del-zota Reverses DMD44 Progression in Phase 1/2 Trial

    PR NEWSWIRE

    Karyopharm Completes Ph3 SENTRY Trial Enrollment in Myelofibrosis

    PR NEWSWIRE

    Gilead Launches New Primary Biliary Cholangitis Campaign

    FIERCE PHARMA

    FDA Approves Sobi`s NASP Biologics License for Uncontrolled Gout

    PR NEWSWIRE

    CONNECTA Advances CTH120 to Ph II for Fragile X Syndrome with EIC

    PR NEWSWIRE

    NICE Thresholds Benefit Ultra-Rare Disease Drugs, Leader Says

    PHARMAWEB

    Ionis Gets FDA Breakthrough Designation for ION582 in Angelman

    BUSINESSWIRE

    Modeyso™ in NCCN® Guidelines for H3 K27M-mutant Diffuse Glioma

    PR NEWSWIRE

    Analog Pharma Canada Debuts First Product in Canadian Market

    PR NEWSWIRE

    FDA Fast Tracks NS-229 for Eosinophilic Granulomatosis Treatment

    PR NEWSWIRE

    Health Canada Approves Bylvay for Alagille Syndrome Treatment

    PR NEWSWIRE

    AstraZeneca India Introduces Eculizumab for aHUS, PNH Treatment

    EXPRESS PHARMA

    Servier Acquires Potential Treatment for Fragile X Syndrome

    PR NEWSWIRE

    Chiesi Highlights Rare Disease Commitment at ICIEM 2025

    GLOBENEWSWIRE

    Capricor Respond to FDA`s Complete Response Letter for Deramiocel

    GLOBENEWSWIRE

    FDA Fast Tracks Teva`s Emrusolmin

    GLOBENEWSWIRE

    JCR Pharma Shares Pabinafusp Alfa`s Data for MPS II at ICIEM

    PHARMAWEB

    AMO Pharma, PHRI, Venca Research License Deal for ARVC Study

    PR NEWSWIRE

    Attralus Unveils Pan-Amyloid Diagnostic Imaging Data at ASNC 2025

    GLOBENEWSWIRE

    FDA Grants NeuroNOS Orphan Drug Status for Glioblastoma in Adults

    GLOBENEWSWIRE

    FDA Issues Complete Response Letter to Saol for SL1009 (DCA)

    PR NEWSWIRE

    BioMarin Reveals Phase 3 Data for in Phenylketonuria Teens

    PR NEWSWIRE

    Tevogen Shares Valuation, Pipeline Forecast on EBV Focus

    GLOBENEWSWIRE

    Walden Advances Pipeline, Supports Ph 2 Kidney Trial

    GLOBENEWSWIRE

    Zevra Reports Data on Miplyffa in Niemann-Pick Disease C

    GLOBENEWSWIRE

    Sangamo Unveils Detailed Data from STAAR Study in Fabry Disease

    GLOBENEWSWIRE

    CorVista Publishes Study on Non-Invasive Pulmonary Test

    GLOBENEWSWIRE

    Pulmovant Gets Orphan Drug Nod in Japan for Mosliciguat

    GLOBENEWSWIRE

    Beacon Reveals 9+ Month DAWN & 36-Month SKYLINE Trial Results

    GLOBENEWSWIRE

    Agios Updates U.S. PDUFA for Pyruknd in Thalassemia

    GLOBENEWSWIRE

    FDA Proposes New Approval Pathway for Rare Diseases

    FIERCE BIOTECH

    Glycomine Starts Global Phase 2b Trial of GLM101 for PMM2-CDG

    PHARMAWEB

    Rallybio Gets $12.5M Equity Milestone from Recursion for REV102

    PHARMAWEB

    Scenic Biotech Reveals Mitochondrial, Cardiac Disease Drug

    GLOBENEWSWIRE

    ZyVersa Data In Alport, DKD Underscore Need For Renal Lipid Drugs

    GLOBENEWSWIRE

    Ministry of Health Designates Assam Medical College & Hospital

    PHARMABIZ

    Ascelia Pharma Submits Orviglance NDA to FDA

    ACCESSWIRE

    Servier Commits up To $530 M Into Eye Cancer Deal with Ideaya

    PRESS RELEASE

    Ascendis Presents Endocrinology Rare Disease Data at ASBMR 2025

    GLOBENEWSWIRE

    Biogen, Stoke Therapeutics Show Zorevunersen Potential in Dravet

    GLOBENEWSWIRE

    Encoded Therapeutics Receives RMAT Designation for ETX101

    PHARMAWEB

    Medipal Holdings, JCR Pharmaceuticals Ink Global Licensing Deal

    PHARMAWEB

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