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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

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    All News #Library
    Rare Diseases

    Kennedy Looks to Fast-Tracking Approvals for Rare Disease Drugs

    REUTERS

    Dyne Therapeutics to Present FSHD Data at FSHD Society Congress

    GLOBENEWSWIRE

    Avadel Gets Orphan Drug Nod for Lumryz in Hypersomnia

    GLOBENEWSWIRE

    Acadia Pharmaceuticals to Host R&D Day in NYC on June 25, 2025

    BUSINESSWIRE

    FDA’s Prasad Vows to Make Rare Disease Drugs Available

    ENDPTS

    ViGeneron Rebrands, Gains FDA RPDD for VG801 in Stargardt

    GLOBENEWSWIRE

    Tempest`s Amezalpat gets Orphan Drug Designation in EU for HCC

    GLOBENEWSWIRE

    Oncovita`s MVdeltaC gets FDA Orphan Drug Designation

    GLOBENEWSWIRE

    Quince Therapeutics Exceeds 75% Enrollment in Phase 3 NEAT Trial

    BUSINESSWIRE

    Affinia Therapeutics and DCM Foundation Partner to Raise Awarenes

    BUSINESSWIRE

    Rilzabrutinib Grants Orphan Designation for Sickle Cell Disease

    PRESS RELEASE

    Sanofi to buy US biopharma group Blueprint for over $9 billion

    PRESS RELEASE

    Ainos` AI Nose Accuracy Breakthrough in Elderly Care Monitoring

    ACCESSWIRE

    Lobe Sciences Names Yong Yao Chief Financial Officer

    ACCESSWIRE

    Spinogenix`s SPG302 for ALS Gets EMA Orphan Drug Designation

    PR NEWSWIRE

    uniQure Updates on AMT-130 for Huntington`s Disease

    GLOBENEWSWIRE

    Protagonist & Takeda`s ASCO Presentation on Ph 3 VERIFY Study

    BUSINESSWIRE

    J Ints Bio to Present Global Clinical Results at ASCO 2025

    PR NEWSWIRE

    LBL-024 Clinical Data in Advanced EP-NEC Revealed at 2025 ASCO

    PR NEWSWIRE

    Entrada Therapeutics Authorized for ELEVATE-45-201 Study in EU

    GLOBENEWSWIRE

    Ainos AI Nose Boosts Hygiene Monitoring Accuracy to 85%

    ACCESSWIRE

    Zydus gets USFDA Fast Track for oral inhibitor Usnoflast

    INDPHARMAPOST

    Stealth to Cut Jobs After FDA Rejects Therapy for Rare Disorder

    REUTERS

    Barth Syndrome Foundation Responds to FDA`s Elamipretide Plan

    BUSINESSWIRE

    Twymeeg Data Presented at 68th Japan Diabetes Society Meeting

    BUSINESSWIRE

    CARsgen`s Satri-cel Granted Priority Review by the NMPA

    PR NEWSWIRE

    New Therapy for Children with Growth Hormone Deficiency Approved

    PR NEWSWIRE

    Taysha Gene Reveals TSHA-102 Trial Design for Rett Syndrome

    GLOBENEWSWIRE

    Rezolute Completes Enrollment in Phase 3 Study of Ersodetug

    GLOBENEWSWIRE

    DEBRA, Dermaliq Partner On Epidermolysis Bullosa Drug Delivery

    GLOBENEWSWIRE

    Biohaven Announces Positive Data for BHV-1400 in IgA Nephropathy

    PR NEWSWIRE

    Sobi to share new clinical data and research at EULAR 2025

    PR NEWSWIRE

    Acadia Pharmaceuticals Appoints Allyson as Senior Vice President

    BUSINESSWIRE

    Alligator Bioscience gets EU Orphan Drug Designation for HLX22

    ACCESSWIRE

    Ambry Genetics Program Impact on Rare Disease Diagnosis

    BUSINESSWIRE

    Moderna to Present at Upcoming Conferences in June 2025

    ACCESSWIRE

    Grin Therapeutics, Angelini Pharma collaborate on Radiprodil

    PR NEWSWIRE

    Rein Therapeutics Begins RENEW Phase 2 Trial of LTI-03 in IPF

    PR NEWSWIRE

    Spur Shows Gene Therapy Proof for FLT201 in Gaucher Diseases

    GLOBENEWSWIRE

    Ocugen Gets Rare Disease Designation For OCU410ST Gene Therapy

    GLOBENEWSWIRE

    Three Factors to Make a Next-Gen eCOA Platform Work

    EXPRESSPHARMA

    SpringWorks Therapeutics gets CHMP approval for Mirdametinib

    GLOBENEWSWIRE

    England`s NICE Recommends Filspari for IgA Nephropathy

    PR NEWSWIRE

    Innate Pharma Reports Durable Lacutamab Results

    BUSINESSWIRE

    Relief Gets FDA Rare Pediatric Tag For RLF-TD011

    ACCESSWIRE

    Soleno Therapeutics Submits EMA Application for Diazoxide Choline

    GLOBENEWSWIRE

    Quoin Pharmaceuticals gets FDA clearance for QRX003 study

    GLOBENEWSWIRE

    Merck Showcases Innovation in Advanced Cancers and Rare Tumors

    BUSINESSWIRE

    PacBio Expands Distribution in China, Accesses New Lab Networks

    GLOBENEWSWIRE

    Telomir Pharmaceutical Raises $3M in Equity Sale for Rare Disease

    ACCESSWIRE

    Dr. Benjamin Greenberg Joins Trethera Scientific Advisory Board

    GLOBENEWSWIRE

    AnnJi Pharma Reports Positive Phase 1/2a results for AJ20

    PR NEWSWIRE

    Sarepta Updates UK Dosing in Elevidys Study for DMD

    BUSINESSWIRE

    Tempus, Verastem Team on CDx for KRAS-Mutant Ovarian Cancer

    BUSINESSWIRE

    Jaguar Starts Phase 2 Trial of Crofelemer for Pediatric MVID

    ACCESSWIRE

    Soleno: VYKAT XR Re-Treatment Eases Prader-Willi Symptoms

    GLOBENEWSWIRE

    Quoin Pharma EMA Grants Orphan Drug Designation for QRX003

    GLOBENEWSWIRE

    SIFI gets Marketing Authorisation for Akantior® in UK

    PR NEWSWIRE

    Applied`s rare disease drug suffers another phase 3 failure

    FIERCE BIOTECH

    Ionis chalks up phase 3 trial win as it works to grow Tryngolza

    FIERCE PHARMA

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