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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    Veranova: A CDMO that manages complexity with confidence.
    All News #Library
    Rare Diseases

    Amylyx Axes Drug Program for Rare Neurodegenerative Disorder

    BIOSPACE

    Travere Gets FDA Nod for REMS Modification on filspari

    BUSINESSWIRE

    Regeneron`s Immune Disorder Therapy Improves Functions in Trial

    REUTERS

    BioVersys Granted EMA Orphan Status for Alpibectir & Ethionamide

    GLOBENEWSWIRE

    Advicenne Publishes Long-term European Study Results of ADV7103

    PHARMAWEB

    Harmony Bio to Unveil Rare Epilepsy Data at Epilepsy Congress

    BUSINESSWIRE

    PharmaTher Updates Post FDA Approval for Ketamine (KETARx™)

    GLOBENEWSWIRE

    Regeneron Reports Ph3 Success in Generalized Myasthenia Gravis

    GLOBENEWSWIRE

    Roca Therapeutics Receives EMA Recognition for Radiation

    PHARMAWEB

    Theravance Bio Completes Enrollment in Pivotal Ph 3 CYPRESS Study

    PR NEWSWIRE

    RNA Biotech Emerges with $52 M and Clinical Rare Disease Asset

    FIERCE BIOTECH

    Baylor Unveils RNA Sequencing Advances for Rare Disease Diagnosis

    GLOBENEWSWIRE

    Arnatar Reveals Orphan Drug, Rare Pediatric Designation for ART4

    GLOBENEWSWIRE

    Coya Therapeutics` IND for COYA 302 in ALS Accepted by FDA

    PR NEWSWIRE

    Lundbeck to Present New Data on Bexicaserin at Upcoming Congress

    PR NEWSWIRE

    Ionis Picks Orsini as Sole Specialty Pharmacy for Dawnzera

    PR NEWSWIRE

    Rusfertide Granted Breakthrough Status for Polycythemia Vera

    PHARMAWEB

    Stealth’s Flight Path Returns to FDA for Rejected Barth Drug

    BIOSPACE

    FDA Gives Nod to 3rd HAE Drug, Ionis` Dawnzera, in 2 Months

    FIERCE PHARMA

    FDA Grant Crinetics Orphan Status for Atumelnant in CAH Treatment

    GLOBENEWSWIRE

    Quoin`s NETHERTON NOW Campaign For Netherton Syndrome Awareness

    GLOBENEWSWIRE

    VeonGen`s VG801 Therapy for Stargardt Gets FDA RMAT Designation

    GLOBENEWSWIRE

    PureTech Unveils Strategy & Ph 2b Data for Deupirfenidone in IPF

    BUSINESSWIRE

    ReAlta Gets Orphan Drug Status for RLS-0071 in Graft-Versus-Host

    BUSINESSWIRE

    GC Biopharma Hunterase Delivery Study Published in SCIE Journal

    PR NEWSWIRE

    NITI Aayog Addresses Rare Diseases Affecting 90M Indians at FICCI

    EXPRESSPHARMA

    Sarepta Extends $700M Debt to 2030 Amid Elevidys Uncertainty

    FIERCE BIOTECH

    Rezolute Recruits Ex-Ultragenyx Exec for Rare Disease Launch

    FIERCE PHARMA

    PTC Therapeutics Receives CRL for Vatiquinone NDA

    PRESS RELEASE

    Keros Gets FDA Orphan Drug Status for KER-065 in DMD

    GLOBENEWSWIRE

    Rezolute Names Sunil Karnawat as Chief Commercial Officer

    GLOBENEWSWIRE

    AlzeCure Livestreams ACD440 Pain Project & Orphan Drug Updates

    PHARMAWEB

    Stealth Bio Submits Rare Disease Application for The Third Time

    PR NEWSWIRE

    Napo Pharma, FDA Discuss Crofelemer for Pediatric MVID Treatment

    PHARMAWEB

    NIH Edits Gene, Fixes Tay-Sachs Mutation in Mice

    FIERCE BIOTECH

    Santhera, Uniphar Agree to Distribute AGAMREE in 5 GCC Countries

    GLOBENEWSWIRE

    US FDA Approves First Therapy for Rare Respiratory Disease

    PR NEWSWIRE

    Tiziana Starts Ph 2a Trial of Intranasal Foralumab in MSA

    PHARMAWEB

    Amber Pharmacy Dispenses BRINSUPRI for Non-CF Bronchiectasis

    BUSINESSWIRE

    Onco360 Adds Modeyso to Oncology, Rare Cancer Therapy Line

    GLOBENEWSWIRE

    VytlOne Forms New Partnership with Insmed

    PR NEWSWIRE

    Insmed Picks PANTHERx Rare to Dispense Brinsupri

    PR NEWSWIRE

    Argenx and Seles Collaborate for Myasthenia Gravis Awareness

    FIERCE PHARMA

    AAVantgarde’s AAVB-039 Gets FDA Fast Track for Stargardt Disease

    GLOBENEWSWIRE

    FDA Approves Jazz Pharma`s Drug for Rare Brain Tumor

    REUTERS

    Ultragenyx Fill Ph3 Aspire Study for GTX-102 in Angelman Syndrome

    GLOBENEWSWIRE

    AZ`s Alexion Highlights gMG in Upcoming Awareness Film

    FIERCE PHARMA

    FDA Approves Alhemo for Hemophilia A/B Treatment in Children

    PR NEWSWIRE

    Ph 2 Trial Shows SGX945`s Efficacy in Treating Behçet`s Disease

    PR NEWSWIRE

    Sanofi’s SAR446523 Gets US Orphan Drug Tag For Multiple Myeloma

    PRESS RELEASE

    Orsini Chosen as PTC Therapeutics` Sephience Pharmacy Partner

    PR NEWSWIRE

    Vivace Therapeutics Gets Orphan Drug Designation for VT3989

    PR NEWSWIRE

    EU Grant Orphan Status to Sumitomo`s Nuvisertib for Myelofibrosis

    PR NEWSWIRE

    AscellaHealth & Abeona Launch Novel Gene Therapy for Rare Disease

    GLOBENEWSWIRE

    Cognition Showcases Zervimesine`s Impact on Dementia & Alzheimer

    GLOBENEWSWIRE

    AbbVie, Gilead, GSK Support Annual Hepatitis Elimination Drive

    FIERCE PHARMA

    CHMP Recommends AQNEURSA For Niemann-Pick Approval In EU

    BUSINESSWIRE

    US FDA approves PTC Therapeutics` Metabolic Disorder Drug

    REUTERS

    Apellis snatches broad kidney Disease Expansion for Empaveli

    FIERCE PHARMA

    Kedrion COAGADEX Gets Orphan Drug Status for aFXD, Begins Trial

    PR NEWSWIRE

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