Ensifentrine is a medication with a novel mechanism of action for the maintenance treatment of chronic obstructive pulmonary disease.
Rare/Orphan Diseases
Posted inCardiology, New Drugs, Rare/Orphan Diseases, Regulatory
Winrevair: Game-Changer in Pulmonary Arterial Hypertension Treatment
Sotatercept is the first drug that actually modifies PAH.
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 inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Duvyzat: Givinostat for Duchenne Muscular Dystrophy Treatment
Oral givinostat delays the progression of DMD.
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.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Roctavian: Gene Therapy for Hemophilia A
A single infusion of valoctocogene roxaparvovec can completely cure severe hemophilia A.