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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    FAMAR CDMO- From early development to commercial manufacturing.
    All News #Library
    Rare Diseases

    Intrabio Gets EU Approval for Aqneursa in Niemann-Pick Type C

    BUSINESSWIRE

    Intrabio Reports Successful Levacetylleucine Trial

    BUSINESSWIRE

    BPGbio Gets FDA Orphan Status for BPM31510 in CoQ10 Deficiency

    GLOBENEWSWIRE

    Keenova Hits 100K Peyronie`s Patients Milestone in Men`s Health

    PR NEWSWIRE

    European Union Approves DAWNZERA For Hereditary Angioedema

    BUSINESSWIRE

    Opna Bio Receives Orphan Drug Designation For OPN-2853

    BUSINESSWIRE

    OSE Immunotherapeutics Welcomes FDA Orphan Designation

    GLOBENEWSWIRE

    Pegrizeprument Receives Orphan Drug Status For Liver Transplant

    PR NEWSWIRE

    Rare Pediatric Disease Vouchers, PBM Reform, Up For Vote In House

    BIOSPACE

    Advicenne: 2026 Financial Calendar

    PHARMIWEB

    Aro Bio To Update Pompe Disease Program At 22nd Worldsymposium

    BUSINESSWIRE

    FDA Grants BPGbio Orphan Status for BPM31510 in CoQ10 Deficiency

    GLOBENEWSWIRE

    Dyne Therapeutics` DYNE-101 Gets Orphan Status in Japan for DM1

    GLOBENEWSWIRE

    FDA Grants Orphan Drug Status to LP-284 for Soft Tissue Sarcoma

    BUSINESSWIRE

    Nanoscope Gets Japan MHLW Sakigake & Orphan Drug Status

    PR NEWSWIRE

    Quoin Pharma Seeks Breakthrough Designation For QRX003

    GLOBENEWSWIRE

    Rein Therapeutics Gets EMA Orphan Drug Status For IPF Candidate

    GLOBENEWSWIRE

    Spur Therapeutics Showcases FLT201 Data For Gaucher Disease

    GLOBENEWSWIRE

    FDA Accepts Sibnayal MAA For dRTA Treatment In US

    PHARMIWEB

    US, EU Approve Telethon`s Waskyra for Wiskott-Aldrich Syndrome

    PHARMIWEB

    Crosswalk Therapeutics Receives National MPS Society Grant

    PR NEWSWIRE

    Atossa Pharma Gets FDA Orphan Status For (Z)-Endoxifen

    PR NEWSWIRE

    Novartis ianalumab receives BTD designation for Sjögren’s disease

    GLOBENEWSWIRE

    Sobi Gets EU Approval For Aspaveli In C3G And Primary IC-MPGN

    PR NEWSWIRE

    JPM Day 3: Rare Disease In The Spotlight

    BIOSPACE

    Santhera Secures Swissmedic Approval for AGAMREE in DMD

    GLOBENEWSWIRE

    Keenova To Showcase Live Dupuytren`s Contracture Treatment

    PR NEWSWIRE

    Genethon Licenses Askbio For Pompe Disease Gene Therapy

    BUSINESSWIRE

    Adolore BioTherapeutics Receives FDA Orphan Drug Designation

    ACCESSWIRE

    FDA Grants Ipsen BTD for IPN60340 in Acute Myeloid Leukemia

    GLOBENEWSWIRE

    Kye Pharma Licenses SLENYTO from Neurim for Canadian Market

    PR NEWSWIRE

    Solid Bio Highlights 2026 Neuromuscular Pipeline For AAV-SLB101

    GLOBENEWSWIRE

    Travere Shares Fall as US FDA Extends Review of Rare Kidney Drug

    REUTERS

    CDER Approved 46 Novel Drugs In 2025, Half For Rare Diseases

    RAPS

    JPM26: Leo Pharma Sharpens Its Eye For Dermatology Dealmaking

    FIERCE PHARMA

    FDA Rejects Atara’s blood Cancer Treatment

    REUTERS

    Alfasigma Acquires Exclusive Rights To Parenteral Adibelivir

    PHARMIWEB

    Orsini Selected as Pharmacy Partner for Cytokinetics`s MYQORZO

    PR NEWSWIRE

    Neurogene Sets 2026 Milestones for NGN-401 in Rett Syndrome

    BUSINESSWIRE

    Novita Pharma Receives FDA Orphan Drug Designation For NP-G2-044

    PR NEWSWIRE

    Precigen Spotlights RRP Therapy Momentum at JP. Morgan Conference

    PR NEWSWIRE

    Servier Sharpens Its Focus On Rare Neurology

    FIERCE BIOTECH

    Solid Biosciences Gets FDA Orphan Status For SGT-212 Gene Therapy

    GLOBENEWSWIRE

    Sentynl Therapeutics Inc. Announces FDA Approval of ZYCUBO

    PRESS RELEASE

    Pierre Fabre Pharma Responds to Fda`s CRL for Tabelecleucel

    PR NEWSWIRE

    Biogen`s High-Dose Spinraza Gets EU Nod for SMA

    GLOBENEWSWIRE

    EU Renews Sibnayal Authorization for Advicenne

    PHARMIWEB

    Agios Details 2026 Strategy To Boost Rare Disease Portfolio

    GLOBENEWSWIRE

    FDA Accepts Pimicotinib NDA For Tenosynovial Giant Cell Tumor

    BUSINESSWIRE

    FDA`s Breakthrough Tag to Encoded`s ETX101 for Dravet Syndrome

    PHARMIWEB

    N-Lorem, Esperare Collaborate To Expand Access To ASO Therapies

    PR NEWSWIRE

    Palvella Therapeutics Provides 2026 Outlook for Rare Skin Disease

    GLOBENEWSWIRE

    SEED Therapeutics Doses 1st Patient in Phase 1 Trial of ST-01156

    GLOBENEWSWIRE

    Aurora Therapeutics Launches Gene Editing for Rare Diseases

    PHARMIWEB

    Amylyx Pharma Nominates Gubra`s AMX0318 as GLP-1RA Candidate

    BUSINESSWIRE

    FDA Accepts AskBio IND for AB-1009 in Pompe Disease LOPD

    GLOBENEWSWIRE

    Encoded Therapeutics Showcases Progress In Dravet Syndrome

    PHARMIWEB

    Nxera Pharma Licenses Vamorolone For Duchenne Muscular Dystrophy

    GLOBENEWSWIRE

    Savara Selects PANTHERx Rare as Exclusive U.S. Specialty Pharmacy

    BUSINESSWIRE

    Tisento Therapeutics Wraps Up Global Phase 2B Zagociguat Study

    GLOBENEWSWIRE

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