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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    Evonik
    All News #Library
    Rare Diseases

    FDA Approves UZEDY® For Bipolar I Disorder In Adults

    GLOBENEWSWIRE

    Quoin Pharma Secures $104.5M Financing In Private Placement

    GLOBENEWSWIRE

    Boehringer Breaks IPF Treatment Drought with New FDA approval

    FIERCE PHARMA

    Aviadobio Secures Option To Rare Eye Disease Gene Therapy

    FIERCE BIOTECH

    Data Reveals Benefit In Infantile-Onset Niemann-Pick Disease

    BUSINESSWIRE

    Genethon Shows 2-Year Efficacy In Duchenne Muscular Dystrophy

    BUSINESSWIRE

    Neurogene To Start NGN-401 Gene Therapy Trial For Rett Syndrome

    BUSINESSWIRE

    Rezolute Issues Inducement Grant Per Nasdaq LR 5635(C)(4)

    GLOBENEWSWIRE

    Optime Care Terminates Distributor Agreement for Korlym

    GLOBENEWSWIRE

    Taysha Gene Therapies Reveals TSHA-102 Data In Rett Syndrome

    GLOBENEWSWIRE

    Alterity Reveals Positive ATH434 Ph 2 Trial Results At Congress

    GLOBENEWSWIRE

    Rein Therapeutics Gets EU Approval To Initiate LTI-03 Ph2 Trial

    GLOBENEWSWIRE

    Quince Therapeutics Showcases Ataxia-Telangiectasia Walking Data

    PHARMIWEB

    Santhera Partners Biomedica For AGAMREE® Distribution In Russia

    GLOBENEWSWIRE

    Ascendis Files MAA to EMA for TransCon CNP in Achondroplasia

    PHARMIWEB

    Abeona, Children`s Hospital Colorado Open ZEVASKYN® Gene Therapy

    GLOBENEWSWIRE

    Ascendis Submits MAA to EMA for TransCon CNP in Achondroplasia

    GLOBENEWSWIRE

    Science 37 Passes Third FDA Inspection for Ph3 Rare Disease Trial

    GLOBENEWSWIRE

    Precision Neuromed Receives FDA Orphan Drug Designation for GBM

    PR NEWSWIRE

    Eyedura Initiates Insulin-Based Neurotrophic Keratitis Treatment

    PHARMIWEB

    Lexeo Therapeutics Advances FDA Talks For Accelerated Approval

    GLOBENEWSWIRE

    Soligenix Reaches Safety Milestone In Phase 3 Trial Of Hybryte™

    PR NEWSWIRE

    Spur Therapeutics Unveils Gaucher Disease Gene Therapy Data

    GLOBENEWSWIRE

    Ryoncil® Revenues Increase 66% In Second Quarter Post Launch

    GLOBENEWSWIRE

    Avidity Bioscience To Present Del-Zota Data In DMD At WMS Society

    PR NEWSWIRE

    Jaguar Health Meets FDA To Advance Crofelemer Trial

    PHARMIWEB

    Myrtelle Share Interim Ph1/2 Gene Therapy Data In Canavan Disease

    PR NEWSWIRE

    Spruce Bio Gets FDA Breakthrough Status For (TA-ERT) In MPS IIIB

    BUSINESSWIRE

    Chiesi, Arbor Weave $2.1B Rare Disease Pact

    BIOSPACE

    Rocket Aborts Mission To Get Rare Disease Gene Therapy Approved

    FIERCE BIOTECH

    CMS Strengthens Orphan Drug Protections From IRA Price

    BIOSPACE

    Aminex Therapeutics Gets FDA Orphan Status For AMXT 1501

    PR NEWSWIRE

    Aavantgarde Secures FDA Orphan Drug Status & UK CTA For AAVB-039

    GLOBENEWSWIRE

    Health Canada Greenlights Agamree For Duchenne DMD

    PR NEWSWIRE

    Lundbeck Presents Amlenetug Phase 3 Mascot Trial Design

    PHARMAWEB

    Savara Presents Phase 3 Impala-2 Trial Results For Molgramostim

    BUSINESSWIRE

    N-Lorem Foundation Hosts 3rd Nano-Rare Patient Colloquium

    BUSINESSWIRE

    Key Milestone In ACTIVATE Phase 2 Clinical Trial Of BIA 28-6156

    PHARMAWEB

    Sobi Choose Kinaxis To Expedite Access To Life-Changing Treatment

    BUSINESSWIRE

    Fortress Bio, Cyprium Therapeutics Update NDA For CUTX-101

    GLOBENEWSWIRE

    Cour Pharma Gets Orphan Status For CNP-106 In Myasthenia Gravis

    GLOBENEWSWIRE

    Pharming Group`s Leniolisib For APDS In Children Accepted By FDA

    GLOBENEWSWIRE

    PNRI Publishes Advances in Rare & Genetic Disease Research

    PR NEWSWIRE

    JCR Pharmaceuticals Announces Licensing Agreement with Menagen

    BUSINESSWIRE

    Opus Sets Sights On FDA Talks After Posting Gene Therapy Data

    GLOBENEWSWIRE

    FDA Grants Expanded Access for Jaguar’s Crofelemer in MVID

    PHARMAWEB

    Abbvie Files BLA For Pivekimab Sunirine To Treat Rare Cancer

    PR NEWSWIRE

    Buloxibutid Granted Orphan Drug Status In Japan For IPF

    ACCESSWIRE

    Disc Medicine Submits Bitopertin NDA For Accelerated EPP Approval

    GLOBENEWSWIRE

    Dyne Pharma Gets Japan Orphan Status For Dyne-251 In DMD

    GLOBENEWSWIRE

    Applied Therapeutics Provides Update Following Meeting With FDA

    GLOBENEWSWIRE

    Larimar Therapeutics Updates Nomlabofusp Program

    GLOBENEWSWIRE

    Tonix Pharma To Launch Prader-Willi Syndrome Ph2 Trial

    GLOBENEWSWIRE

    Japan Grants Orphan Drug Designation to JR-446 for MPS IIIB

    BUSINESSWIRE

    Savara Unveils Phase 3 Impala-2 Trial Data On Molgramostim

    BUSINESSWIRE

    Evkeeza Approval For Ultra-Rare Pediatric High Cholesterol

    GLOBENEWSWIRE

    Givinostat Data Revealed For Duchenne Muscular Dystrophy At 2025

    PR NEWSWIRE

    CSL Vifor, Travere Therapeutics Acknowledge New KDIGO Guidelines

    PR NEWSWIRE

    Orsini Chosen As Crinetics` Palsonify Specialty Pharmacy Partner

    PR NEWSWIRE

    FDA Grants Orphan Drug Status To Biostar Pharma`s Utidelone

    PR NEWSWIRE

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