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Discovery/Preclinical
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Novel discovery in gene therapy to treat kidney diseases …
Newer Targets
Stanford researchers program cancer-fighting cells to resist exhaustion, attack solid tumors…
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Dyno Therapeutics Announces Research Published in Science Enabling Artificial Intelligence Approach…
Newer Targets
Hepion Pharmaceuticals’ CRV431 Prevents Cirrhosis in Experimental Model of Severe Liver…
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FDA Committee Votes 6-6 on Durect’s Non-Opioid Pain Drug
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Novo Nordisk’s Ozempic Wins Approval for Reducing Cardiovascular Events in Diabetes…
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FDA advisory panel split on whether to approve Durect’s Posimir for…
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Orphan-paediatric
Orphan-paediatric
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Orphan-paediatric
Wave shares sink on disappointing results for WVE-120102 in Huntington’s disease trial
Firstwordpharma News
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January 2, 2020
Orphan-paediatric
Alnylam’s Lumasiran Sees Positive Results for Ultra-Rare Kidney Disease
Orphan-paediatric
Homology Medicines Announces Encouraging Initial Clinical Data from its pheNIX Gene Therapy Trial for PKU
Orphan-paediatric
Alnylam’s RNAi therapy lumasiran hits main goal of late-stage study in primary hyperoxaluria type 1
Orphan-paediatric
FORMA Therapeutics Announces Clinical Data for Sickle Cell Disease Candidate FT-4202, Demonstrating Multi-modal Activity of PKR Activation Increases Oxygen Affinity and Decreases Sickle Hemoglobin...
Orphan-paediatric
HIZENTRA (Immune Globulin Subcutaneous [Human] 20% Liquid) Receives Orphan-Drug Exclusivity for...
Firstwordpharma News
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December 9, 2019
0
Orphan-paediatric
Marinus Announces Ganaxolone Orphan Epilepsy Program Updates Including Planned Clinical Program...
Firstwordpharma News
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December 9, 2019
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Orphan-paediatric
ASH19: Pfizer, Sangamo’s haemophilia A gene therapy SB-525 continues to show...
Firstwordpharma News
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December 9, 2019
0
Orphan-paediatric
ASH19: Serious vaso-occlusive crises eliminated in some sickle cell disease patients...
Firstwordpharma News
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December 9, 2019
0
Orphan-paediatric
3DBio Therapeutics Receives Orphan Drug Designation for AuriNovo for Ear Reconstruction...
Firstwordpharma News
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November 19, 2019
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