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Rare/Orphan Diseases

Kimmtrak: Groundbreaking Immuno-Oncological Treatment for Metastatic Uveal Melanoma
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

Kimmtrak: Groundbreaking Immuno-Oncological Treatment for Metastatic Uveal Melanoma

January 27, 2022September 8, 2022

Tebentafusp is a pharmacological breakthrough in the fight against a deadly disease.

Welireg: New Treatment for Tumors in Von Hippel–Lindau Disease
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

Welireg: New Treatment for Tumors in Von Hippel–Lindau Disease

January 26, 2022September 8, 2022

Belzutifan by Merck & Co. is for the treatment of renal cell carcinoma, central nervous system hemangioblastomas, and pancreatic neuroendocrine tumors.

Biogen to Develop Updated Version of Spinraza
Posted inNeuroscience, Pharma & Biotech, Rare/Orphan Diseases

Biogen to Develop Updated Version of Spinraza

January 5, 2022September 8, 2022

Nusinersen for the treatment of spinal muscular atrophy will get a follow-up.

Leqvio: Newest Drug Against ‘Very Bad’ Cholesterol
Posted inMetabolism, New Drugs, Rare/Orphan Diseases, Regulatory

Leqvio: Newest Drug Against ‘Very Bad’ Cholesterol

December 23, 2021September 8, 2022

Two injections of inclisiran a year and you can forget about high cholesterol. Novartis doesn’t lie.

Vyvgart: New Drug to Treat Myasthenia Gravis
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Vyvgart: New Drug to Treat Myasthenia Gravis

December 19, 2021September 8, 2022

Efgartigimod developed by Argenx is a strong competitor to Soliris.

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.

dwarf family
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Voxzogo: First Drug to Treat Dwarfism

December 6, 2021September 8, 2022

Vosoritide by BioMarin Pharmaceutical may cure achondroplasia.

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