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Rare/Orphan Diseases

Posted inClinical Trials, Metabolism, Neuroscience, Rare/Orphan Diseases

Redasemtide: Universal Medicine for Regeneration of Any Tissue

by Nik Resovski and Timur Burkhanayev February 12, 2022September 8, 2022

Redasemtide as a lifesaver for the most devastating diseases, including stroke, osteoarthritis, cardiomyopathy, and bullous epidermolysis.

Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Enjaymo: First Drug to Treat Cold Agglutinin Disease

by Julia Mardi February 9, 2022September 8, 2022

Sutimlimab will reduce the need for red blood cell transfusion due to hemolysis.

Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Tavneos: New Drug for ANCA-Associated Vasculitis

by Tanya von Reuss February 5, 2022September 8, 2022

Avacopan is better than prednisone in the treatment of granulomatosis with polyangiitis and microscopic polyangiitis.

Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

Kimmtrak: Groundbreaking Immuno-Oncological Treatment for Metastatic Uveal Melanoma

by Tanya von Reuss January 27, 2022September 8, 2022

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

Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

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

by Tanya von Reuss 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.

Posted inNeuroscience, Pharma & Biotech, Rare/Orphan Diseases

Biogen to Develop Updated Version of Spinraza

by Julia Mardi January 5, 2022September 8, 2022

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

Posted inCardiology, Metabolism, New Drugs, Rare/Orphan Diseases, Regulatory

Leqvio: Newest Drug Against ‘Very Bad’ Cholesterol

by Mark Gubar December 23, 2021April 5, 2024

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

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