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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

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    All News #Library
    Rare Diseases

    argenx progresses ARGX-119 for Congenital Myasthenic Syndromes

    GLOBENEWSWIRE

    Riliprubart Wins Japan Orphan Drug Tag for CIDP Therapy

    PRESS RELEASE

    FDA Approves Gamifant for MAS in Still’s Disease Patients

    PR NEWSWIRE

    Schrödinger Gets Fast Track for SGR-1505 in Macroglobulinemia

    BUSINESSWIRE

    Ultragenyx Earns Breakthrough Status for GTX-102 in Angelman

    GLOBENEWSWIRE

    Orphalan partners with MAP International and Wilson Disease Assoc

    GLOBENEWSWIRE

    Scinai Immuno’s PC111 Wins Top Italian Dermatology Award

    PR NEWSWIRE

    Grace Therapeutics Submits New Drug Application for GTx-104

    GLOBENEWSWIRE

    Ovid Therapeutics Enters Agreement with Immedica Pharma

    GLOBENEWSWIRE

    FDA Grants RPD Tag to QRX003 for Netherton Syndrome

    GLOBENEWSWIRE

    PharmaTher Secures Key US Patent for Ketamine in ALS

    GLOBENEWSWIRE

    Abeona Ph 3 RDEB Data Published in The Lancet Journal

    GLOBENEWSWIRE

    Jaguar Health Extends Maturity Date of Convertible Notes to Jan

    ACCESSWIRE

    Jaguar Health Updates Orphan Disease Intestinal Failure Program

    ACCESSWIRE

    Cumberland Pharmaceuticals Shares Phase 2 FIGHT DMD Trial Results

    PR NEWSWIRE

    Sephience (Sepiapterin) Approved by European Commission for PKU

    PR NEWSWIRE

    Cure Rare Disease Gets FDA Feedback on LGMD2I Gene Therapy

    BUSINESSWIRE

    Arcturus Therapeutic to Host KOL Presentation on ARCT-810 Phase 2

    BUSINESSWIRE

    Dupixent US Approval For Bullous Pemphigoid Treatment

    PRESS RELEASE

    SpringWorks Therapeutics gets CHMP nod for Nirogacestat

    GLOBENEWSWIRE

    Cycle Pharma Harliku Tablets FDA-Approved for Alkaptonuria

    BUSINESSWIRE

    UCB presents TK2d research at UMDF 2025 Conference

    PR NEWSWIRE

    Larimar Sets Call on Nomlabofusp for Friedreich’s Ataxia

    GLOBENEWSWIRE

    Argenx gets EU approval for Vyvgart injection for CIDP

    GLOBENEWSWIRE

    Capricor Therapeutics: Positive 4-Year Data from HOPE-2 Study

    GLOBENEWSWIRE

    Saol Therapeutics Poster at UMDF Mitochondrial Med. Conf. 2025

    PR NEWSWIRE

    DEA Judge Exposes Bias in Cannabis Grower Licensing Case

    ACCESSWIRE

    Chiesi Global Rare Diseases Awards Research Grants for Lysosomal

    BUSINESSWIRE

    Paradigm Therapeutics Receives Investment to Develop SD-101

    PR NEWSWIRE

    Camurus` POSITANO study: CAM2029 effects in liver disease

    PR NEWSWIRE

    BPGbio Presents Preliminary Clinical Data for BPM31510

    GLOBENEWSWIRE

    Senti Bio`s SENTI-202 Granted FDA Orphan Drug Designation for AML

    GLOBENEWSWIRE

    Diagonal’s DIAG723 Antibody Gets ODD from U.S. FDA and EMA

    GLOBENEWSWIRE

    Telomir Pharmaceuticals Prevents Cellular Aging in Progeria

    ACCESSWIRE

    JCR Pharmaceuticals Launches MPS Type II Disease Awareness Film

    BUSINESSWIRE

    Actio Biosciences Raises $66M for Genetics-Driven Therapeutics

    BUSINESSWIRE

    FDA Grants Orphan Designation To Medicovestor ADC For Cancer

    GLOBENEWSWIRE

    Capricor Gets Orphan Drug Designation For BMD, Advances DMD

    GLOBENEWSWIRE

    Lobe Advances Psilocin After Spinout Deal With Cynaptec

    ACCESSWIRE

    RemeGen`s Telitacicept (RC18) Received Orphan Drug Designation

    PR NEWSWIRE

    Azitra Reports Positive Ph1b Data In Netherton Syndrome

    PR NEWSWIRE

    Sarepta Updates ELEVIDYS Safety for Non-Ambulatory DMD

    BUSINESSWIRE

    US FDA approves Nuvation Bio`s rare lung cancer drug

    REUTERS

    Angitia Biopharmaceuticals Announces Topline Results of AGA2115

    GLOBENEWSWIRE

    Novartis Fabhalta® improves hemoglobin in new PNH patients

    GLOBENEWSWIRE

    Blueprint Medicines: Data on AYVAKIT® Treatment at 2025 Congresse

    PR NEWSWIRE

    EU Commission Grants Orphan Drug Status to Florbetaben (18F)

    PR NEWSWIRE

    Sumitomo Pharma America: Nuvisertib (TP-3654) Gets FDA Fast Track

    PR NEWSWIRE

    Vascarta gets FDA Orphan Drug Designation for Vasceptor

    PR NEWSWIRE

    Mezzion Expands Udenafil Pipeline to Target ADPKD Kidney Market

    PR NEWSWIRE

    Ouro Medicines to Trial OM336 for Autoimmune Cytopenias

    PR NEWSWIRE

    Sobi shares new clinical data on hematologic diseases at EHA 2025

    PR NEWSWIRE

    Incyte’s Retifanlimab Phase 3 Data in SCAC Published in Lancet

    BUSINESSWIRE

    Ionis CDO Dr Geary to Retire Dr Kordasiewicz Named Successor

    BUSINESSWIRE

    ENYO Pharma Completes Series C Financing with Vesalius Biocapital

    BUSINESSWIRE

    Travere to Present Filspari Data at Podocyte Conference

    BUSINESSWIRE

    First Patient Dosed in Ph 3 Study of ION582 in Angelman syndrome

    BUSINESSWIRE

    Avidity Biosciences to Present Phase 1/2 Fortitude Trial Data

    PR NEWSWIRE

    FDA Grants Orphan Drug Tag to Veverimer for Anti-GBM

    PR NEWSWIRE

    Vanda Pharmaceuticals: First Patient Dosed in VCA-894A Trial

    PR NEWSWIRE

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