Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.
EMA
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 inInfections, New Drugs, Regulatory
Beyfortus: Effective Protection for Infants Against Respiratory Syncytial Virus
One injection of nirsevimab reliably protects newborns against the development of RSV-associated lower respiratory tract infections.
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.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Zynteglo: Gene Therapy for Beta-thalassemia
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Tecvayli: New Drug for Multiple Myeloma
Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.
Posted inAutoimmune Diseases, Clinical Trials, In the Pipeline, New Drugs, Regulatory
Alopecia Areata Treatment: What Future Holds
What new drugs for hair loss are being developed by the pharmaceutical industry?
