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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

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    All News #Library
    Rare Diseases

    Dyne Files BLA for Z-Rostudirsen in Exon 51 Duchenne MD

    GLOBENEWSWIRE

    NanoViricides: NV-387 Ready for DRC Ebola Emergency Deployment

    PHARMIWEB

    Prilenia Donates Trial Data to CHDI for Huntington`s Research

    BUSINESSWIRE

    Regenxbio, Novartis & Dyne Near FDA Filing for Muscular Dystrophy

    BIOSPACE

    BioLineRx and Hemispherian Highlight GLIX1 Data at ASCO 2026

    PR NEWSWIRE

    CHMP Issues Positive Opinion for Nerandomilast in IPF and PPF

    GLOBENEWSWIRE

    Enable Injections Gets CE Certification for enFuse with Aspaveli

    GLOBENEWSWIRE

    Servier Showcases Rare Oncology Portfolio Growth at ASCO 2026

    PR NEWSWIRE

    Insurers Better Prepared for Rare Disease Outbreaks Post-COVID-19

    BUSINESSWIRE

    Agios`Pyrukynd Approved in EU for Adults with Thalassemia

    GLOBENEWSWIRE

    First medicine to treat rare uncontrolled growth of body tissues

    EMA

    Tyra Creates Awareness Day to Shine Light on a Rare Cancer

    FIERCE PHARMA

    Mirum Pharma to Present Rare Liver Disease Data at EASL 2026

    BUSINESSWIRE

    Amgen Updates Tavneos Label In Japan Amid Liver Injury Review

    FIERCE PHARMA

    Avalyn to Present AP01 Pulmonary Fibrosis Data at EULAR 2026

    GLOBENEWSWIRE

    BioMarin Bounces Back as Voxzogo Beats Expectations

    BIOSPACE

    Oddifact Granted EU Orphan Drug Status For Infliximab

    GLOBENEWSWIRE

    Telomir Pharma Publishes Telomir-Zn Data in Wilson`s Disease

    PHARMIWEB

    Palvella Presents QTORIN Data for Rare Vascular Malformations

    GLOBENEWSWIRE

    Savara Presents IMPALA-2 Biomarker Data on Molgramostim at ATS

    BUSINESSWIRE

    Zydus Lifesciences Eyes Specialty and Rare Disease Acquisitions

    ECONOMICTIMES

    BioMarin`s Drug Show Significant Growth Gains in Children

    REUTERS

    Jaguar Health Reports First Quarter 2026 Financials

    PHARMIWEB

    CureDuchenne Partners with Tevard for DMD tRNA Therapy

    PR NEWSWIRE

    Jaguar Health`s Crofelemer Trial Continues in Pediatric MVID

    PHARMIWEB

    Cullinan Therapeutics Gets FDA Orphan Designation for CLN-049

    GLOBENEWSWIRE

    CLS Health`s Dr. Chhabra Helps Child with Rare Conditions Walk

    ACCESSWIRE

    CLS Health Helps Child with Three Rare Conditions Walk Normally

    PHARMIWEB

    Amgen Backs Tavneos Despite Japan Liver Toxicity Reports

    ENDPOINTS

    Design Therapeutics Reports Friedreich`s Ataxia Data Readout

    ENDPOINTS

    BioLineRx and Hemispherian: GLIX1 Shows Anti-Tumor Effect in GBM

    BIOSPACE

    FDA Grants INTERACT Meeting for Glafabra`s Fabry Disease Program

    FIRSTWORD PHARMA

    NICU Genetic Testing Market Powers Personalized Patient Outcomes

    PHARMIWEB

    Oligomerix Completes Ph 1A Clinical Study On OLX-07010

    PHARMIWEB

    Sciensus and Disc Medicine Showcase Rare Disease Survey Framework

    FIRSTWORD PHARMA

    Eton Pharma Acquires U.S. Rights to IMPAVIDO (miltefosine)

    GLOBENEWSWIRE

    Lundbeck Receives Japan Orphan Drug Status for Asedebart in CAH

    PR NEWSWIRE

    Sanofi Rare Lung Disease Drug Outperforms Standard Care in Trial

    REUTERS

    BioMarin Updates Phase 3 Trial of BMN 401 in Children with ENPP1

    PR NEWSWIRE

    ImmunityBio Secures Patents for ANKTIVA+BCG Cancer Combo to 2035

    PHARMIWEB

    Belite Bio Gets Orphan Drug Status for Tinlarebant in Switzerland

    GLOBENEWSWIRE

    JASCAYD (Nerandomilast) Approved in Japan for IPF & PPF Treatment

    GLOBENEWSWIRE

    Krystal Biotech Gets UK MHRA Approval for VYJUVEK in EB Treatment

    GLOBENEWSWIRE

    FDA Alignment Is Critical for Rare Disease; AI Can Help

    BIOSPACE

    Teva: Only 23% of Young Adults with TD Symptoms Are Diagnosed

    GLOBENEWSWIRE

    Liavium-CA1 Pregabalin Gets FDA Conditional OK for Canine Pain

    PR NEWSWIRE

    Amylyx Publishes Phase 2 HELIOS Trial Data for AMX0035

    PRESS RELEASE

    MHRA Approves Vyjuvek for Dystrophic Epidermolysis Bullosa

    PRESS RELEASE

    Amgen`s Rare Disease Drug Linked to Deaths with- liver injuries

    REUTERS

    FDA Places Clinical Hold on Aardvark`s Drug for Extreme Hunger

    REUTERS

    FDA Approves New CRYSVITA Dosing Option for Adults with XLH

    GLOBENEWSWIRE

    Cognition Therapeutics Gets Donation to Extend CT1812 Access

    GLOBENEWSWIRE

    HCW Biologics: HCW11-040 Prevents Bronchopulmonary Dysplasia

    GLOBENEWSWIRE

    Regenxbio Hits Duchenne Milestone, Eyes 2027 FDA Approval

    BIOSPACE

    Polaryx Therapeutics Wins GHP Healthcare Pharma Award

    GLOBENEWSWIRE

    Mezzion Secures Financing to Advance Phase 3 FUEL-2 Trial

    PR NEWSWIRE

    Biocryst To Unveil Pediatric Hereditary Angioedema Impact Data

    GLOBENEWSWIRE

    Varda, United Therapeutics Collaborate on Rare Pulmonary Therapy

    PR NEWSWIRE

    BridgeBio Submits Encaleret NDA to FDA for ADH1 Patients

    GLOBENEWSWIRE

    Agios Unveils Mitapivat Data for Rare Anemias at EHA 2026

    GLOBENEWSWIRE

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