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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    Munit offers scalable GMP micronization and particle size engineering to enhance drug solubility, bioavailability, and performance.
    All News #Library
    Rare Diseases

    Quoin Pharma Sets Up Japan Unit to Prep QRX003 for Market

    GLOBENEWSWIRE

    Chiesi Rare Diseases Showcases Lipodystrophy Progress at ENDO

    GLOBENEWSWIRE

    Denali Sells Rare Pediatric Priority Review Voucher for $195M

    GLOBENEWSWIRE

    SK Pharmteco Partners with Orphan Therapeutics for Rare Disease

    GLOBENEWSWIRE

    Constantiam and Cincinnati Children`s Team Up on Gaucher Disease

    GLOBENEWSWIRE

    From fragile skin to living medicine: Lessons from RDEB

    BIOSPACE

    ConSynance Doses First Patient in CSTI-500 Phase 2a PWS Trial

    GLOBENEWSWIRE

    Mabion Revives MabionCD20 via Rare Disease Deal with Oddifact

    INDPHARMAPOST

    Quoin Pharma Reports Positive Update in Netherton Syndrome CUP

    GLOBENEWSWIRE

    Sanofi`s Tzield Approved in US as First Disease-Modifying therapy

    PRESS RELEASE

    Amgen Challenges Regulator Proposal to Withdraw Tavneos Approval

    FIRSTWORD PHARMA

    Lundbeck drug candidate shows promise in Cushing`s disease trial

    REUTERS

    Rhythm Pharma Shows Setmelanotide Phase 2 Interim Data at ENDO

    GLOBENEWSWIRE

    Novartis Says Rare Muscle Disease Drug Shows Promise in Trial

    REUTERS

    Amgen Shores Up Tavneos` FDA Defense with Duke Data Analysis

    BIOSPACE

    Belite Bio Completes NDA Rolling Submission for Tinlarebant

    GLOBENEWSWIRE

    BioCryst Presents New HAE Data at 2026 EAACI Annual Meeting

    GLOBENEWSWIRE

    Jaguar Health to Present Crofelemer Updates on June 24

    ACCESSWIRE

    Novartis RemIND Data Show Rhapsido Potential for CIndU

    GLOBENEWSWIRE

    Rhythm Pharma to Report Setmelanotide Phase 2 Prader-Willi Data

    GLOBENEWSWIRE

    Rocket Pharma Closes $180M Sale of Priority Review Voucher

    BUSINESSWIRE

    O`Shaughnessy Ventures Backs Bioengineer`s Own-Disease Cure

    PR NEWSWIRE

    HUTCHMED Highlights Sovleplenib Phase III Data at EHA 2026

    GLOBENEWSWIRE

    J&J Eyes Rare Disease Expansion for Imaavy After Trial Win

    FIERCE PHARMA

    Quetzal Doses First Patient in QTX-2101 Phase 3 APL Trial

    GLOBENEWSWIRE

    Rein Therapeutics Expands Phase 2 RENEW Trial into United Kingdom

    GLOBENEWSWIRE

    Sobi to Present New Clinical Data at EHA 2026 in Stockholm

    PR NEWSWIRE

    Novel Gene Therapy Restores Muscle Function in DMD Models

    MDANDERSON

    Egetis` MCT8 Episode Re-Airs on Lifetime MCT8 Deficiency

    ACCESSWIRE

    Italfarmaco Gets Health Canada Priority Review for Givinostat

    GLOBENEWSWIRE

    Servier, n-Lorem Foundation Partner on Rare Disease Research

    PR NEWSWIRE

    Disc`s FDA Meeting Sets Path for Embattled Rare Blood Drug

    BIOSPACE

    Rare disease biotech Beren adds $300M from new financings

    ENDPOINTS

    Tris Pharma Gets FDA Orphan Drug Status for TRN-257 Oxybate

    GLOBENEWSWIRE

    Facial Pain Association Marks One-Year of Pain Registry

    GLOBENEWSWIRE

    Beren Therapeutics Secures $300M for Niemann-Pick Disease Type C

    BUSINESSWIRE

    Spruce Shows Long-Term Tralesinidase Alfa ERT Data in MPS IIIB

    BUSINESSWIRE

    Teva Showcases AUSTEDO Long-Term Advances in Tardive Dyskinesia

    GLOBENEWSWIRE

    Alkeus Doses First Patient in Phase 3 Gildeuretinol Study

    GLOBENEWSWIRE

    Zevra Therapeutics Lists Patent to Protect MIPLYFFA Exclusivity

    GLOBENEWSWIRE

    Busy FDA: Complementary Pathways Despite Unanswered Questions

    BIOSPACE

    Post-Makary, New Rare Disease Framework Faces Fresh Uncertainty

    BIOSPACE

    Ambros Therapeutics Doses First Patient in CRPS-RISE Ph 3 Trial

    GLOBENEWSWIRE

    Ascendis to Highlight Rare Endocrine Disease Progress at ENDO`26

    GLOBENEWSWIRE

    NanoViricides Proposes Phase 2 Oral Ebola Treatment Trial

    ACCESSWIRE

    Neurocrine Highlights INGREZZA Efficacy in Tardive Dyskinesia

    PR NEWSWIRE

    Neurogene Completes Dosing in NGN-401 Rett Syndrome Trial

    BUSINESSWIRE

    Incyte Eyes $2B Deal for Star to Grow Haematology Pipeline

    REUTERS

    Grifols Backs AlphaDetect to Speed Alpha-1 Antitrypsin Detection

    PR NEWSWIRE

    MHRA Conducts Benefit-Risk Review of Avacopan

    PRESS RELEASE

    Chiesi Wins EU Approval of LOJUXTA® Capsules for Paediatric HoFH

    GLOBENEWSWIRE

    IntraBio`s EMA Application for Aqneursa in Ataxia-Telangiectasia

    BUSINESSWIRE

    Acting FDA chief meets with rare disease groups to mend fences

    REUTERS

    Pharming Receives FDA Acceptance of sNDA Resubmission for Joenja

    GLOBENEWSWIRE

    Regeneron to Highlight Metabolic, Ophthalmic, Rare Disease at ADA

    GLOBENEWSWIRE

    Acting FDA Commissioner Pledges to Prevent Political Interference

    ENDPOINTS

    Jazz Pharmaceuticals to Present Xywav Data at SLEEP 2026

    PR NEWSWIRE

    Quoin Pharma Gets Japan MHLW Orphan Designation for QRX003

    GLOBENEWSWIRE

    Biogen`s Salanersen Gets FDA Breakthrough Designation for SMA

    GLOBENEWSWIRE

    Travere Presents Long-Term DUPLEX Phase 3 Data at ERA 2026

    BUSINESSWIRE

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