Sotatercept is the first drug that actually modifies PAH.
FDA
Posted inMetabolism, New Drugs, Rare/Orphan Diseases, Regulatory
Lenmeldy/Libmeldy: Most Expensive Drug in the World
Atidarsagen autotemcel is a gene therapy for metachromatic leukodystrophy (MLD).
Posted inCardiology, Metabolism, New Drugs, Regulatory
Tryvio: Brand New High Blood Pressure Medication
Aprocitentan for the treatment of resistant arterial hypertension.
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Duvyzat: Givinostat for Duchenne Muscular Dystrophy Treatment
Oral givinostat delays the progression of DMD.
Posted inNew Drugs, Oncology, Regulatory
Tevimbra: Immuno-Oncologic Treatment for Esophageal Cancer
Tislelizumab for the treatment of unresectable or metastatic esophageal squamous cell carcinoma.
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Relyvrio/Albrioza: New Drug for Amyotrophic Lateral Sclerosis Treatment
Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy
A single dose of the elivaldogene autotemcel will cure a deadly disease.