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  • Topic
    • COVID-19
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    • Metabolism
    • Neuroscience
    • Autoimmune Diseases
    • Infections
    • Rare/Orphan Diseases
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brain
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

Relyvrio/Albrioza: New Drug for Amyotrophic Lateral Sclerosis Treatment

September 29, 2022September 30, 2022

Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.

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

Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy

September 19, 2022September 19, 2022

A single dose of the elivaldogene autotemcel will cure a deadly disease.

Beyfortus: Effective Protection for Infants Against Respiratory Syncytial Virus
Posted inInfections, New Drugs, Regulatory

Beyfortus: Effective Protection for Infants Against Respiratory Syncytial Virus

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

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

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

August 24, 2022September 8, 2022

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

alopecia areata
Posted inAutoimmune Diseases, Clinical Trials, In the Pipeline, New Drugs, Regulatory

Alopecia Areata Treatment: What Future Holds

August 3, 2022September 8, 2022

What new drugs for hair loss are being developed by the pharmaceutical industry?

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