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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

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    All News #Library
    Rare Diseases

    Pfizer-Valneva Lyme disease shot shows more than 70% efficacy in

    REUTERS

    Genentech Drug Fails Rare Disease Trial, Raises Obesity Risk

    FIERCE BIOTECH

    Rhythm Pharma Names PANTHERx Rare Exclusive U.S. Specialty

    PR NEWSWIRE

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

    GLOBENEWSWIRE

    Zevra Therapeutics Grants Inducement to New CFO Under Nasdaq

    GLOBENEWSWIRE

    N-Lorem Provides Insight On Commercializing Nano-Rare Medicines

    PHARMIWEB

    FDA Approves IMCIVREE for Acquired Hypothalamic Obesity

    GLOBENEWSWIRE

    Sarepta Eyes FDA Nod for Duchenne Exon Skippers Despite Trial

    BIOSPACE

    Shionogi Enrolls First Patients In Global Phase 2 Trial

    PHARMIWEB

    Soligenix Announces HyBryte Clinical Summary Published

    PR NEWSWIRE

    After Alfasigma licensing deal, Lynavoy picks up FDA nod

    FIERCE PHARMA

    Emapalumab`s EMBRACE Phase 2A Results In Interferon-Gamma Sepsis

    PHARMIWEB

    Glaukos Announces Commercial Availability of Epioxa

    PHARMIWEB

    Stoke Aims To Restore Developmental Loss In Dravet Syndrome

    FIERCE BIOTECH

    Rare Disease Advocates Stage `funeral` at FDA, Make 4 Demands

    FIERCE BIOTECH

    Sanofi’s Venglustat Earns BTD in US for Type 3 Gaucher Disease

    GLOBENEWSWIRE

    Mirecule Expands Strategic FSHD Collaboration With Sanofi

    PHARMIWEB

    FDA Rejects Aldeyra Eye Disease Candidate for 3rd Time

    FIERCE BIOTECH

    FDA Fast-Tracks Polaryx`s PLX-200 for Late-Infantile CLN2 Disease

    GLOBENEWSWIRE

    New Two-Year APPROACH Data Show Durable TransCon CNP Benefits

    GLOBENEWSWIRE

    Palvella Pharma Secures European Patent For Rapamycin

    GLOBENEWSWIRE

    Denali’s Hunter Syndrome Drug Gains Focus After REGENXBIO Setback

    BIOSPACE

    Rhythm Pharma Reveals Phase 3 EMANATE Trial Topline Results

    GLOBENEWSWIRE

    Sentynl Therapeutics Enters into Agreement with PRG S&T

    PR NEWSWIRE

    Solve GNE Advances GNE Myopathy Program Toward Phase 1 Trial

    GLOBENEWSWIRE

    Zevra Pharma Sells SDX Portfolio To Commave Pharma For $50M

    GLOBENEWSWIRE

    Shilpa Biologicals And Mabtree Biologics Secure ODD From USFDA

    INDPHARMAPOST

    Bridgebio`s Muscle Weakness Data Impress Ahead Of FDA Filing

    PRESS RELEASE

    Ultragenyx Gene Therapy Cuts Ammonia in Phase 3 Trial

    FIERCE BIOTECH

    Inovio Trims Team As Trouble Brews At FDA For Rare Disease Drug

    FIERCE BIOTECH

    Polaryx Therapeutics Strengthens Krabbe Disease Community

    GLOBENEWSWIRE

    Regenxbio Gene Therapy Passes Interim Test As Pivotal Data Loom

    FIERCE BIOTECH

    Quoin Pharma Gets FDA Fast Track For QRX003 In Netherton Syndrome

    GLOBENEWSWIRE

    Genomics Reveals Underdiagnosis Of Rare Genetic Disorders

    PR NEWSWIRE

    Small Molecules Are `Resurgent` In Orphan Drug R&D: Evaluate

    FIERCE BIOTECH

    Aminex`s 2nd ODD for AMXT 1501 in Malignant Glioma Including DIPG

    PR NEWSWIRE

    BioMarin Presents VOXZOGO Achondroplasia Data at 2026 ACMG Meet

    PR NEWSWIRE

    Capricor Therapeutics Announces Late-Breaking HOPE-3 Data

    GLOBENEWSWIRE

    FDA approves leucovorin for Rare Genetic Disorder Causing Autism

    REUTERS

    Senator Ron Launch Investigation Into FDA Rare Disease Rejection

    BIOSPACE

    Capricor Therapeutics Sets New PDUFA Date For Deramiocel BLA

    GLOBENEWSWIRE

    BBP-418 Shows Efficacy in Ph3 FORTIFY Study in LGMD2I/R9

    GLOBENEWSWIRE

    Regenxbio Unveils Positive Interim Data from RGX-202 Phase I/II

    PR NEWSWIRE

    Solid Bio Provide Interim Positive Clinical Update on Ph1/2 Trial

    GLOBENEWSWIRE

    Rare Disease Patients Can`t Wait For Regulatory Process

    BIOSPACE

    Andelyn Biosciences Launches Andelyn Foundation For Rare Disease

    PR NEWSWIRE BIO

    Dyne To Pursue FDA Approval Following Positive Long-Term Data

    BIOSPACE

    Eledon`s Tegoprubart Wins Orphan Status for Liver Transplants

    GLOBENEWSWIRE

    Mipletamig Shows 86% Clinical Benefit Rate With No CRS In AML

    PHARMIWEB

    Satellos Unveils SAT-3247 Data In Duchenne Muscular Dystrophy

    GLOBENEWSWIRE

    Soligenix Gains MHRA PIM Designation for SGX945

    PR NEWSWIRE

    Tenaya Therapeutics Showcases Tn-301`s Promise In Duchenne

    GLOBENEWSWIRE

    MDA 2026 Spotlights Brogidirsen`s 4.5-Year DMD Data

    PR NEWSWIRE

    Abeona Therapeutics Boosts ZEVASKYN Launch Momentum

    GLOBENEWSWIRE

    Chiesi Global and Protalix Announce EU Approval Elfabrio

    GLOBENEWSWIRE

    GENFIT Secures FDA Orphan Drug Designation for NTZ in ACLF

    GLOBENEWSWIRE

    Immedica Names Orsini Exclusive Specialty Pharmacy for LOARGYS

    PR NEWSWIRE

    ITF Presents New Givinostat Analyses for DMD at MDA 2026

    PR NEWSWIRE

    AGAMREE Shows Long-Term Safety, Efficacy in Duchenne Disease

    GLOBENEWSWIRE

    Spruce Hooks A Commercial Chief To Prep For Rare Disease Launch

    FIERCE PHARMA

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