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Mirum`s rare liver Disease Drug Meets Main Goal in Mid-Stage
05 May 2026 //
REUTERS
Rhythm Pharma to Share Hypothalamic Obesity Data at PES
04 May 2026 //
GLOBENEWSWIRE
US FDA Grants Orphan Drug Designation To NV-387 For Measles
04 May 2026 //
PHARMIWEB
Feinstein Finds Two Paths to Fetal Anemia, New Drug Targets
04 May 2026 //
FIRSTWORD PHARMA
Neurocrine Survey Reveals Tardive Dyskinesia`s Toll on Jobs
04 May 2026 //
PR NEWSWIRE
Kubota Inks Deal With Laboratoires KÔL For Stargardt Disease
04 May 2026 //
PHARMIWEB
Pharma Industry Wants Clarity On Plausible Mechanism Framework
04 May 2026 //
RAPS
Ascidian Wraps Dose Escalation In ACDN-01 For Stargardt Disease
04 May 2026 //
PR NEWSWIRE
Opus Genetics Gets FDA Acceptance of OPGx-LCA5 Into RDEP Program
04 May 2026 //
FIRSTWORD PHARMA
Atossa Gets FDA Rare Pediatric Tag for Endoxifen in MAS Syndrome
04 May 2026 //
PR NEWSWIRE
Satellite Gets FDA Rare Pediatric Disease Designation for SB-101
04 May 2026 //
PHARMIWEB
Kedrion Highlights Rare Disease Work on Plasminogen Awareness Day
04 May 2026 //
PR NEWSWIRE
Spur Therapeutics Unveils FLT201 for Adult Type 1 Gaucher Disease
04 May 2026 //
GLOBENEWSWIRE
Palvella Initiates Phase2 For QTORIN Rapamycin for Angiokeratomas
04 May 2026 //
GLOBENEWSWIRE
Biomarin Reveals VOXZOGO Impact On Achondroplasia In Children
03 May 2026 //
PR NEWSWIRE
Novartis Gets Health Canada Nod For Fabhalta In Adult C3G
02 May 2026 //
FIRSTWORD PHARMA
EU Approves IMCIVREE for Acquired Hypothalamic Obesity
01 May 2026 //
FIRSTWORD PHARMA
Arrowhead Gets TGA Approval for Plozasiran in FCS in Australia
01 May 2026 //
FIRSTWORD PHARMA
SPARC To Sell Rare Pediatric Disease Priority Review Voucher
01 May 2026 //
EXPRESSPHARMA
Larimar backs skin biomarker in Friedreich’s ataxia
30 Apr 2026 //
FIRSTWORD PHARMA
LEO Pharma Strengthens Rare Skin Disease Focus With Replay Gene
30 Apr 2026 //
PHARMIWEB
Chiesi Snaps Up Kalvista For $1.9B, Betting Big On Rare Disease
30 Apr 2026 //
BIOSPACE
AstraZeneca Cuts Asthma and Rare Disease Units; Boosts Cancer
30 Apr 2026 //
BIOSPACE
Norgine Announces European Commission Marketing Authorisation
29 Apr 2026 //
PR NEWSWIRE
Patient Advocate Gives Recursion a Reason to Fight Rare Disease
29 Apr 2026 //
BIOSPACE
Expression Therapeutics Secures Rare FDA Approval for Hemophilia
29 Apr 2026 //
PR NEWSWIRE
Remepy Collaborates With Merck KGaA To Advance Hybrid Drugs
28 Apr 2026 //
PR NEWSWIRE
BRC Therapeutics Clears FDA For BRC-002 IND Application
28 Apr 2026 //
PR NEWSWIRE
Pantherx Rare Pharma Wins 2026 MMIT Patient Choice Award
28 Apr 2026 //
PR NEWSWIRE
Solid Biosciences Receives EU Orphan Drug Status For SGT-003
28 Apr 2026 //
GLOBENEWSWIRE
Rocket Pharma Announces $180 M Sale of Priority Review Voucher
28 Apr 2026 //
PHARMIWEB
Santhera Reveals AIFA Board Approval For Agamree Reimbursement
28 Apr 2026 //
GLOBENEWSWIRE
Soligenix Reports Interim Phase 3 FLASH2 HyBryte Results
28 Apr 2026 //
PR NEWSWIRE
FDA Proposes to withdraw Amgen`s drug for Rare Autoimmune Disease
27 Apr 2026 //
REUTERS
Hope Rises For Rare Gynaecological Clear Cell Cancer
24 Apr 2026 //
FIRSTWORD PHARMA
Ultragenyx Issues Inducement Grant Per Nasdaq LR 5635(C)(4)
24 Apr 2026 //
GLOBENEWSWIRE
Prasad`s FDA Exit Aids Rare Diseases; New CBER Head Restores
24 Apr 2026 //
BIOSPACE
Frankfurt Court Upholds Besremi Arbitral Award For AOP Health
24 Apr 2026 //
FIRSTWORD PHARMA
EU Backs Arrowhead’s REDEMPLO for Rare Lipid Disorder
24 Apr 2026 //
BUSINESSWIRE
FDA Approves Regeneron`s First Gene Therapy for Hearing Loss
24 Apr 2026 //
FDA
Polaryx Therapeutics Joins National Tay-Sachs Conference
23 Apr 2026 //
GLOBENEWSWIRE
AlphaRose`s RareLabs Launches AI Platform, Names Freishtat CEO
23 Apr 2026 //
FIRSTWORD PHARMA
Capricor Therapeutics Unveils HOPE-3 Phase 3 Results At AAN 2026
22 Apr 2026 //
GLOBENEWSWIRE
Denali CEO Celebrates Launch Of Rare Disease Drug
22 Apr 2026 //
BIOSPACE
Ionis Antisense Drug Stabilizes Ultra-Rare Disease Before FDA
22 Apr 2026 //
BIOSPACE
Ojemda Approved 1st EU Targeted Therapy in Pediatric LLG Glioma
22 Apr 2026 //
GLOBENEWSWIRE
Dupixent Approval For Children With Chronic Spontaneous Urticaria
22 Apr 2026 //
PRESS RELEASE
Sanofi`s Tzield FDA Approved To Delay Stage 3 Type 1 Diabetes
22 Apr 2026 //
PRESS RELEASE
Pegrizeprument Secures Orphan Status in Heart Transplant
22 Apr 2026 //
PR NEWSWIRE
Astra eyes 5th Ultomiris indication after kidney disease trial
22 Apr 2026 //
PRESS RELEASE
Roche’s Enspryng cuts relapse risk by 68% in Neuroinflammatory
22 Apr 2026 //
PRESS RELEASE
Polaryx Wins FDA Fast Track for All Four SOTERIA Indications
21 Apr 2026 //
GLOBENEWSWIRE
Duchenne Innovation Thrives For Real Progress
21 Apr 2026 //
BIOSPACE
Star Therapeutics Secures FDA RPD, BTD for VGA039 in VWD
21 Apr 2026 //
PHARMIWEB
Ionis Shares Pivotal Zilganersen Data in Alexander Disease
21 Apr 2026 //
BUSINESSWIRE
Pasithea Therapeutics Announces Grant of RPDD by FDA to PAS-004
20 Apr 2026 //
GLOBENEWSWIRE
Medicus Pharma Seeks FDA Orphan Drug Designation For Skinject
19 Apr 2026 //
FIRSTWORD PHARMA
Monopar Reveals ALXN1840 Outperforms SoC In Wilson Disease
19 Apr 2026 //
GLOBENEWSWIRE
EU Approves Merck`s ENFLONSIA for Infant RSV Prevention
17 Apr 2026 //
PHARMIWEB
Drug Farm To Unveil Positive Phase 1B Results For ALPK1
17 Apr 2026 //
PHARMIWEB
