Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.
Regulatory
Actions of government agencies regulating the pharmaceutical industry: approval, rejection, and prohibition of drugs, granting special statuses.
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 inAutoimmune Diseases, New Drugs, Regulatory
Sotyktu: Miracle Pill for Autoimmune Diseases
Deucravacitinib by Bristol-Myers Squibb can help with psoriasis, psoriatic arthritis, Crohn’s disease, ulcerative colitis, and systemic lupus erythematosus.
Posted inCOVID-19, Infections, New Drugs, Regulatory
Pfizer and Moderna Release Booster Vaccines to Protect Against Omicron
Updated bivalent mRNA vaccines Comirnaty and Spikevax target Omicron subvariants BA.4 and BA.5.