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gene therapy

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.

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.

Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis

December 9, 2021September 8, 2022

The combination drug will help almost all cystic fibrosis patients.

Vyjuvek: Effective Treatment for Dystrophic Epidermolysis Bullosa
Posted inClinical Trials, Rare/Orphan Diseases

Vyjuvek: Effective Treatment for Dystrophic Epidermolysis Bullosa

November 30, 2021September 8, 2022

Krystal Biotech’s beremagene geperpavec gene therapy will save from imminent death.

Bit Bio: Mass and Problem-Free Production of Any Human Cells
Posted inAutoimmune Diseases, Neuroscience, Oncology, Pharma & Biotech

Bit Bio: Mass and Problem-Free Production of Any Human Cells

November 11, 2021September 8, 2022

Human cells are needed for scientific research, development of new drugs, and creation of cell therapies.

Zolgensma: Gene Therapy That Cure Spinal Muscular Atrophy. The Most Comprehensive Review
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

Zolgensma: Gene Therapy That Cure Spinal Muscular Atrophy. The Most Comprehensive Review

April 8, 2021September 8, 2022

Novartis revealed the most expensive drug in the world. And it is amazing.

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