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  • Topic
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    • Infections
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Rare/Orphan Diseases

Alsitek: New Drug for ALS
Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases

Alsitek: New Drug for ALS

June 17, 2022September 8, 2022

Oral masitinib doubles the life in patients with amyotrophic lateral sclerosis.

olorofim
Posted inClinical Trials, Infections, Rare/Orphan Diseases

Olorofim: New Antifungal Drug

June 9, 2022September 8, 2022

Olorophim by F2G and Shionogi & Co. is focused on invasive aspergillosis treatment.

Tepezza: First Drug for Thyroid Eye Disease
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Tepezza: First Drug for Thyroid Eye Disease

April 27, 2022September 12, 2022

Teprotumumab by Horizon Therapeutics can really help with proptosis and diplopia in Graves’ disease and beyond.

lymphatic vessels
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Vijoice: First Targeted Treatment for PIK3CA-Related Overgrowth Spectrum (PROS)

April 23, 2022September 8, 2022

Novartis and its alpelisib will help with very rare diseases with malformations and excessive tissue growth.

Redasemtide: Universal Medicine for Regeneration of Any Tissue
Posted inClinical Trials, Metabolism, Neuroscience, Rare/Orphan Diseases

Redasemtide: Universal Medicine for Regeneration of Any Tissue

February 12, 2022September 8, 2022

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

Enjaymo: First Drug to Treat Cold Agglutinin Disease
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Enjaymo: First Drug to Treat Cold Agglutinin Disease

February 9, 2022September 8, 2022

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

Tavneos: New Drug for ANCA-Associated Vasculitis
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory

Tavneos: New Drug for ANCA-Associated Vasculitis

February 5, 2022September 8, 2022

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

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