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FDA`s Orphan Drug Director Out Amid IRA Exemption Rollout
16 Oct 2025 //
BIOSPACE
Azitra Joins Dermatologic Rare Disease Panel At Growth Summit
15 Oct 2025 //
PR NEWSWIRE
Baylor Genetics to Share RNA Seq Rare Disease Data at ASHG
15 Oct 2025 //
GLOBENEWSWIRE
Belite Bio Gets Priority Review From China NMPA For Tinlarebant
15 Oct 2025 //
GLOBENEWSWIRE
Illumina Tech Reveals Hidden Genomic Insights in GeneDx Study
15 Oct 2025 //
PR NEWSWIRE
Minovia Pharma Granted FDA Orphan Drug Designation For MNV-201
15 Oct 2025 //
GLOBENEWSWIRE
Novo Nordisk Pens $2.1B Deal For Omeros` Stalled PNH Drug
15 Oct 2025 //
FIERCE BIOTECH
Transpire Bio Receives FDA Orphan Drug Designation For 2 Diseases
15 Oct 2025 //
GLOBENEWSWIRE
Boehringer Ingelheim Picks Orsini as Jascayd Specialty Pharmacy
14 Oct 2025 //
PR NEWSWIRE
FINTEPLA (OLE) Study Results In Lennox-Gastaut Syndrome Published
14 Oct 2025 //
PR NEWSWIRE
Inmune Bio Joins Dermatologic Rare Disease Panel at Growth Summit
14 Oct 2025 //
GLOBENEWSWIRE
Brogidirsen 3.5Y Trial Shows Results in Duchenne MD at WMS 2025
14 Oct 2025 //
PR NEWSWIRE
Rocket Pharma`s Kresladi BLA Resubmission Accepted By FDA
14 Oct 2025 //
PHARMIWEB
Excimer Laser Shows Promise In Treating Mycosis Fungoides Variant
14 Oct 2025 //
GLOBENEWSWIRE
Biocryst Expands Rare Diseases Focus In $700M Astria Deal
14 Oct 2025 //
FIERCE BIOTECH
Denali`s Hunter Syndrome Drug Latest To Be Hit By FDA Delay
14 Oct 2025 //
FIERCE BIOTECH
PANTHERx Rare Selected by Chiesi for Distribution of Mycapssa
13 Oct 2025 //
PR NEWSWIRE
Annji Showcases Clinical Breakthrough Of AJ201 At WMS 2025
13 Oct 2025 //
PR NEWSWIRE
FDA Approves UZEDY® For Bipolar I Disorder In Adults
10 Oct 2025 //
GLOBENEWSWIRE
Quoin Pharma Secures $104.5M Financing In Private Placement
10 Oct 2025 //
GLOBENEWSWIRE
Boehringer Breaks IPF Treatment Drought with New FDA approval
09 Oct 2025 //
FIERCE PHARMA
Aviadobio Secures Option To Rare Eye Disease Gene Therapy
09 Oct 2025 //
FIERCE BIOTECH
Data Reveals Benefit In Infantile-Onset Niemann-Pick Disease
09 Oct 2025 //
BUSINESSWIRE
Genethon Shows 2-Year Efficacy In Duchenne Muscular Dystrophy
09 Oct 2025 //
BUSINESSWIRE
Neurogene To Start NGN-401 Gene Therapy Trial For Rett Syndrome
09 Oct 2025 //
BUSINESSWIRE
Rezolute Issues Inducement Grant Per Nasdaq LR 5635(C)(4)
09 Oct 2025 //
GLOBENEWSWIRE
Optime Care Terminates Distributor Agreement for Korlym
09 Oct 2025 //
GLOBENEWSWIRE
Taysha Gene Therapies Reveals TSHA-102 Data In Rett Syndrome
09 Oct 2025 //
GLOBENEWSWIRE
Alterity Reveals Positive ATH434 Ph 2 Trial Results At Congress
09 Oct 2025 //
GLOBENEWSWIRE
Rein Therapeutics Gets EU Approval To Initiate LTI-03 Ph2 Trial
09 Oct 2025 //
GLOBENEWSWIRE
Quince Therapeutics Showcases Ataxia-Telangiectasia Walking Data
09 Oct 2025 //
PHARMIWEB
Santhera Partners Biomedica For AGAMREE® Distribution In Russia
09 Oct 2025 //
GLOBENEWSWIRE
Ascendis Files MAA to EMA for TransCon CNP in Achondroplasia
09 Oct 2025 //
PHARMIWEB
Abeona, Children`s Hospital Colorado Open ZEVASKYN® Gene Therapy
08 Oct 2025 //
GLOBENEWSWIRE
Ascendis Submits MAA to EMA for TransCon CNP in Achondroplasia
08 Oct 2025 //
GLOBENEWSWIRE
Science 37 Passes Third FDA Inspection for Ph3 Rare Disease Trial
08 Oct 2025 //
GLOBENEWSWIRE
Precision Neuromed Receives FDA Orphan Drug Designation for GBM
07 Oct 2025 //
PR NEWSWIRE
Eyedura Initiates Insulin-Based Neurotrophic Keratitis Treatment
07 Oct 2025 //
PHARMIWEB
Lexeo Therapeutics Advances FDA Talks For Accelerated Approval
07 Oct 2025 //
GLOBENEWSWIRE
Soligenix Reaches Safety Milestone In Phase 3 Trial Of Hybryte™
07 Oct 2025 //
PR NEWSWIRE
Spur Therapeutics Unveils Gaucher Disease Gene Therapy Data
07 Oct 2025 //
GLOBENEWSWIRE
Ryoncil® Revenues Increase 66% In Second Quarter Post Launch
06 Oct 2025 //
GLOBENEWSWIRE
Avidity Bioscience To Present Del-Zota Data In DMD At WMS Society
06 Oct 2025 //
PR NEWSWIRE
Jaguar Health Meets FDA To Advance Crofelemer Trial
06 Oct 2025 //
PHARMIWEB
Myrtelle Share Interim Ph1/2 Gene Therapy Data In Canavan Disease
06 Oct 2025 //
PR NEWSWIRE
Spruce Bio Gets FDA Breakthrough Status For (TA-ERT) In MPS IIIB
06 Oct 2025 //
BUSINESSWIRE
Chiesi, Arbor Weave $2.1B Rare Disease Pact
06 Oct 2025 //
BIOSPACE
Rocket Aborts Mission To Get Rare Disease Gene Therapy Approved
03 Oct 2025 //
FIERCE BIOTECH
CMS Strengthens Orphan Drug Protections From IRA Price
03 Oct 2025 //
BIOSPACE
Aminex Therapeutics Gets FDA Orphan Status For AMXT 1501
02 Oct 2025 //
PR NEWSWIRE
Aavantgarde Secures FDA Orphan Drug Status & UK CTA For AAVB-039
02 Oct 2025 //
GLOBENEWSWIRE
Health Canada Greenlights Agamree For Duchenne DMD
02 Oct 2025 //
PR NEWSWIRE
Lundbeck Presents Amlenetug Phase 3 Mascot Trial Design
02 Oct 2025 //
PHARMAWEB
Savara Presents Phase 3 Impala-2 Trial Results For Molgramostim
02 Oct 2025 //
BUSINESSWIRE
N-Lorem Foundation Hosts 3rd Nano-Rare Patient Colloquium
02 Oct 2025 //
BUSINESSWIRE
Key Milestone In ACTIVATE Phase 2 Clinical Trial Of BIA 28-6156
02 Oct 2025 //
PHARMAWEB
Sobi Choose Kinaxis To Expedite Access To Life-Changing Treatment
02 Oct 2025 //
BUSINESSWIRE
Fortress Bio, Cyprium Therapeutics Update NDA For CUTX-101
01 Oct 2025 //
GLOBENEWSWIRE
Cour Pharma Gets Orphan Status For CNP-106 In Myasthenia Gravis
01 Oct 2025 //
GLOBENEWSWIRE
Pharming Group`s Leniolisib For APDS In Children Accepted By FDA
01 Oct 2025 //
GLOBENEWSWIRE
