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FDA approves leucovorin for Rare Genetic Disorder Causing Autism
11 Mar 2026 //
REUTERS
Senator Ron Launch Investigation Into FDA Rare Disease Rejection
11 Mar 2026 //
BIOSPACE
Capricor Therapeutics Sets New PDUFA Date For Deramiocel BLA
11 Mar 2026 //
GLOBENEWSWIRE
Andelyn Biosciences Launches Andelyn Foundation For Rare Disease
10 Mar 2026 //
PR NEWSWIRE BIO
Dyne To Pursue FDA Approval Following Positive Long-Term Data
10 Mar 2026 //
BIOSPACE
Eledon`s Tegoprubart Wins Orphan Status for Liver Transplants
10 Mar 2026 //
GLOBENEWSWIRE
Satellos Unveils SAT-3247 Data In Duchenne Muscular Dystrophy
10 Mar 2026 //
GLOBENEWSWIRE
FDA official calls UniQure`s Huntington`s disease treatment a fai
06 Mar 2026 //
REUTERS
Seastar Medical Completes FDA Enrollment For QUELIMMUNE
05 Mar 2026 //
GLOBENEWSWIRE
Sonothera Showcases RIPPLE For DMD At MDA 2026
05 Mar 2026 //
PHARMIWEB
Biogen Presents New SMA Data at MDA, SMA Europe Conferences
05 Mar 2026 //
GLOBENEWSWIRE
Drug Farm`s DF-003 Accepted By FDA For ROSAH Syndrome Evidence
05 Mar 2026 //
PHARMIWEB
Tanabe Pharma America Showcases ALS Research At MDA 2026
05 Mar 2026 //
PR NEWSWIRE
Bayer Ditches 1 AAV Gene Therapy From Pompe Disease Pair
04 Mar 2026 //
FIERCE BIOTECH
MHRA & NHS Ethics Approval Received for ENDOCAN Phase 2 Trial
04 Mar 2026 //
PR NEWSWIRE
Clearmind Advances to Next Trial Phase on DSMB Nod
04 Mar 2026 //
GLOBENEWSWIRE
Transposon Therapeutics To Study TPN-101 For Healthy Aging
04 Mar 2026 //
GLOBENEWSWIRE
Sanofi`s Rilzabrutinib Granted Orphan Drug Status In Japan
03 Mar 2026 //
PRESS RELEASE
Theravance Biopharma Misses Phase 3 CYPRESS Goal, Cuts Costs
03 Mar 2026 //
PR NEWSWIRE
Chiesi Global Rare Diseases Gets FDA Nod For JUXTAPID
03 Mar 2026 //
GLOBENEWSWIRE
Northsea Therapeutics Reaches Key Regulatory Milestones
03 Mar 2026 //
BUSINESSWIRE
Priovant`s Brepocitinib Nda For Dermatomyositis Accepted By FDA
03 Mar 2026 //
GLOBENEWSWIRE
Rein Therapeutics Initiates Phase 2 Trial For LTI-03
03 Mar 2026 //
GLOBENEWSWIRE
Acadia Pushes For Re-Review Of CHMP`s Daybue Snub
03 Mar 2026 //
FIERCE PHARMA
ReviR Doses First in Ph1, Gains CDE Orphan Nod
02 Mar 2026 //
PR NEWSWIRE
Cure Rare and LGMD2L Foundation Announce Multi-Year Partnership
02 Mar 2026 //
PHARMIWEB
Spinogenix Secures FRAXA Backing For Phase 2B (FXS) Trial
02 Mar 2026 //
PR NEWSWIRE
Lumicera Secures URAC Rare Disease Pharmacy Center Of Excellence
02 Mar 2026 //
BUSINESSWIRE
A Pastor’s Journey Finding Right Care and Treatment for NF1-PN
02 Mar 2026 //
GLOBENEWSWIRE
Rhythm Pharma Reveals More Positive Phase 3 Setmelanotide Data
01 Mar 2026 //
GLOBENEWSWIRE
FDA approves Novo`s Sogroya as the first and only FDA Approval
28 Feb 2026 //
PR NEWSWIRE
FDA Approves Once-Weekly YUVIWEL for Children with Achondroplasia
28 Feb 2026 //
GLOBENEWSWIRE
FDA Approve Once-Weekly Yuviwel for Children Aged 2 Years & Older
27 Feb 2026 //
GLOBENEWSWIRE
FDA Approves Biomarin`s PALYNZIQ For Adolescents With PKU
27 Feb 2026 //
PR NEWSWIRE
Illumina Accelerates Rare Disease Testing In Florida
27 Feb 2026 //
PR NEWSWIRE
Santhera Proposes AGAMREE® Inclusion In Spain`s Health System
27 Feb 2026 //
GLOBENEWSWIRE
Rare Groups Push for Speed as Makary Defends Strict Stance
27 Feb 2026 //
BIOSPACE
EMA CHMP Backs X4`s Mavorixafor Approval for EU WHIM Syndrome
27 Feb 2026 //
GLOBENEWSWIRE
Makary Upholds FDA`s Rare Disease Rejection Decisions
27 Feb 2026 //
BIOSPACE
Ipsen Receives Positive CHMP Opinion for Ojemda
27 Feb 2026 //
GLOBENEWSWIRE
Norgine Gets Positive CHMP Opinion for Mavorixafor in EU
27 Feb 2026 //
PR NEWSWIRE
Polaryx Reaffirms Commitment to Rare Pediatric Disorders
27 Feb 2026 //
GLOBENEWSWIRE
DNDi, Sanofi Get CHMP Nod for Single-Dose Sleeping Sickness Tx
27 Feb 2026 //
PRESS RELEASE
Santhera Proposes AGAMREE Inclusion In Spain`s Health System
27 Feb 2026 //
GLOBENEWSWIRE
Quotient Sciences Extends Mfg Partnership For Ultra-Rare Disease
27 Feb 2026 //
PRESS RELEASE
Aardvark Voluntary Pauses HERO Trial in Prader-Willi Syndrome
27 Feb 2026 //
GLOBENEWSWIRE
Evox Therapeutics, RSRT Partner on ExoEdit for Rett Syndrome
26 Feb 2026 //
PHARMIWEB
Neurogene Reveals FDA Breakthrough For NGN-401 In Rett Syndrome
26 Feb 2026 //
BUSINESSWIRE
Netherton Now Spotlights Netherton Syndrome on Rare Disease Day
26 Feb 2026 //
GLOBENEWSWIRE
Sarepta to Present Gene Therapy, Exon-Skipping Data at MDA 2026
26 Feb 2026 //
BUSINESSWIRE
Soligenix SGX945 Gets EMA Orphan Designation for Behçet`s Disease
26 Feb 2026 //
PR NEWSWIRE
GEMMABio Announces First Patient Dosed in CHARISMA Trial of GB221
26 Feb 2026 //
PR NEWSWIRE
Senate Hearing Takes Aim At FDA`s Rare Disease Review Process
26 Feb 2026 //
FIERCE PHARMA
Johnson & Johnson Seeks FDA Approval For IMAAVY In wAIHA Anemia
25 Feb 2026 //
PR NEWSWIRE
Rare Disease Drug Approvals Spur Pantherx Research On Care
25 Feb 2026 //
PR NEWSWIRE
FDA approves Eton`s liquid therapy for rare water-balance
25 Feb 2026 //
REUTERS
FDA Accepts Vanda`s BLA for Imsidolimab in GPP Psoriasis
25 Feb 2026 //
PR NEWSWIRE
Azafaros Publishes Nizubaglustat Data for GM2 Gangliosidosis
25 Feb 2026 //
BUSINESSWIRE
Cashing In On `Creative Fundamentals` To Fund Rare Disease R&D
25 Feb 2026 //
BIOSPACE
Cumberland Pharmaceuticals Launches U.S. Promotion of Talicia
25 Feb 2026 //
PR NEWSWIRE
