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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    DRL offers a portfolio of products & services, including APIs, CMO services, generics, biosimilars & differentiated formulations.
    All News #Library
    Rare Diseases

    Abeona Sells Rare Pediatric Disease Voucher for $155 Million

    GLOBENEWSWIRE

    Belite Bio Completes Enrollment in Global Phase 3 PHOENIX Trial

    GLOBENEWSWIRE

    Apellis to Get $300M from Royalty Deal with Sobi for Aspaveli

    GLOBENEWSWIRE

    Syntis Bio secures $38M to speed up oral therapies for obesity

    BUSINESSWIRE

    Sarcoidosis Research Foundation Launches Enhanced Data Registry

    GLOBENEWSWIRE

    Tilde Sciences Launches VECAMYL™ Through Optime Care

    GLOBENEWSWIRE

    argenx progresses ARGX-119 for Congenital Myasthenic Syndromes

    GLOBENEWSWIRE

    Riliprubart Wins Japan Orphan Drug Tag for CIDP Therapy

    PRESS RELEASE

    Esteve to Acquire Medicine to Treat Medullary Thyroid Cancer

    PR NEWSWIRE

    Iksuda Therapeutics Receives FDA IND Clearance for IKS014

    BUSINESSWIRE

    Arcturus Reports Positive Ph 2 Data in OTC Deficiency

    BUSINESSWIRE

    Rocket Pharma Gets FDA IND Approval for RP-A701 in DCM

    BUSINESSWIRE

    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

    Jaguar Health to Add Crypto to Treasury Reserves

    ACCESSWIRE

    SineuGene`s SNUG01 Receives FDA Orphan Drug Designation for ALS

    PR NEWSWIRE

    Biodexa activates first site for Phase 3 Serenta trial in FAP

    GLOBENEWSWIRE

    Soleno Therapeutics Presents New VYKAT(TM) XR Data

    GLOBENEWSWIRE

    Riliprubart Earns Orphan Drug Status in Us for Organ Rejection

    GLOBENEWSWIRE

    Grace Therapeutics Submits New Drug Application for GTx-104

    GLOBENEWSWIRE

    Ovid Therapeutics Enters Agreement with Immedica Pharma

    GLOBENEWSWIRE

    Orphan Drug Designation for JR-446 for MPS Type IIIB by EC

    BUSINESSWIRE

    TNF Pharma & DADA2 Start Compassionate Use Study of Isomyosamine

    BUSINESSWIRE

    Sephience Gets EU OK for PKU Drug Sepiapterin

    PR NEWSWIRE

    FDA Grants RPD Tag to QRX003 for Netherton Syndrome

    GLOBENEWSWIRE

    Lundbeck gets Orphan drug status in US and EU for Lu AG13909

    PR NEWSWIRE

    PharmaTher Secures Key US Patent for Ketamine in ALS

    GLOBENEWSWIRE

    Abeona Ph 3 RDEB Data Published in The Lancet Journal

    GLOBENEWSWIRE

    Sterotherapeutics Starts Phase 2 Trial for Cushing`s Syndrome

    PR NEWSWIRE

    Jaguar Health Extends Maturity Date of Convertible Notes to Jan

    ACCESSWIRE

    SciSparc Announces 1-for-21 Reverse Share Split

    GLOBENEWSWIRE

    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

    Chiesi Global Awards Grants to Advance Lysosomal Disease Research

    GLOBENEWSWIRE

    Cure Rare Disease Gets FDA Feedback on LGMD2I Gene Therapy

    BUSINESSWIRE

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

    BUSINESSWIRE

    Larimar Therapeutics Gets FDA Feedback on Nomlabofusp BLA Details

    GLOBENEWSWIRE

    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

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