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EMA

gene therapy
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy

by Tanya von Reuss September 19, 2022September 19, 2022

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

by Julia Mardi September 17, 2022September 20, 2022

One injection of nirsevimab reliably protects newborns against the development of RSV-associated lower respiratory tract infections.

blood gene therapy
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Roctavian: Gene Therapy for Hemophilia A

by Julia Mardi September 13, 2022September 13, 2022

A single infusion of valoctocogene roxaparvovec can completely cure severe hemophilia A.

blood
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Zynteglo: Gene Therapy for Beta-thalassemia

by Mark Gubar September 13, 2022September 13, 2022

One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.

multiple-myeloma
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

Tecvayli: New Drug for Multiple Myeloma

by Mark Gubar August 24, 2022September 8, 2022

Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.

Posted inClinical Trials, Infections, New Drugs, Regulatory

Hepcludex: Effective Treatment for Chronic Hepatitis D

by Julia Mardi July 18, 2022September 8, 2022

Bulevirtide is the only approved antiviral drug against hepatitis delta.

Posted inCOVID-19, Infections, New Drugs, Regulatory

Covovax/Nuvaxovid: Nanoparticle Vaccine for COVID-19

by Mark Gubar July 14, 2022April 11, 2024

Covovax/Nuvaxovid by Novavax is a protein subunit vaccine from virus-like particles.

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