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Pharma & Biotech

Why Praxis’ latest FDA win could matter far beyond one genetic epilepsy program

by Soujanya Ravi
June 22, 2026
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Find out how Praxis’ FDA Breakthrough Therapy Designation for elsunersen could reshape SCN2A-DEE epilepsy treatment and ASO development.

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Latest news and features

Alfasigma’s Nordic Pharma deal is more than a bolt-on: why the €190m business could reshape its specialty-care strategy
What do BEL512 Phase 2 results mean for Belenos Biosciences and CRSwNP treatment?
Can a multi-drug longevity protocol really slow functional aging? AgelessRx just won $1m to test it
Silence Therapeutics raised $175m right after SANRECO success. What does it unlock for divesiran?
AN2 Therapeutics is betting on three Phase 2 programs, but can its boron pipeline deliver after epetraborole’s MAC setback?
Three antibiotics, three different AMR battles: why Innoviva’s Prix Galien triple could matter beyond the awards
Restylane Shaype has cleared Europe, but can Galderma turn its “bone-mimicking” filler into a new chin-shaping standard?
VCN-01 moves toward Phase 2/3 retinoblastoma trial after Theriva completes study design
LB-102 pivotal trial accelerates as LB Pharmaceuticals expands psychiatric pipeline
FDA endorsement moves once-weekly sutacimig into decisive Glanzmann thrombasthenia study
SEP-479 clears first pharmacokinetic hurdle in pursuit of oral hypoparathyroidism therapy
Tenax heart failure drug fails Phase 3 test despite 49% drop in cardiac stress biomarker

POPULAR NEWS

Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

Why Mirum Pharmaceuticals and Incyte’s zilurgisertib data could reset expectations in FOP treatment

June 15, 2026

Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

Niagen wins twin rare-disease designations, but can NB4168 turn nicotinamide riboside evidence into a medicine?

July 14, 2026

Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

Gilead and Merck pipeline update reveals promise in HIV and pressure in oncology

June 9, 2026

Latest business and industry news

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Colombia earthquake death toll reaches 224 as rescuers search for thousands missing

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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