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Rare/Orphan Diseases

Ultomiris (ravulizumab).
Posted inAutoimmune Diseases, Rare/Orphan Diseases, Regulatory

Ultomiris: First Drug for Paroxysmal Nocturnal Hemoglobinuria in Children and Adolescents

by Julia Mardi June 11, 2021September 8, 2022

Alexion Pharmaceuticals’ ravulizumab has expanded the range of indications for treating a rare complement-mediated disease.

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

Empaveli: Powerful New Drug for Paroxysmal Nocturnal Hemoglobinuria

by Tanya von Reuss and Alex Avisoni May 20, 2021September 8, 2022

Pegcetacoplan is more effective in treating paroxysmal nocturnal hemoglobinuria compared to Soliris.

Alexion Pharmaceuticals.
Posted inAutoimmune Diseases, Mergers & Acquisitions, Rare/Orphan Diseases

AstraZeneca + Alexion = Rescue of Both Companies

by Alex Avisoni April 18, 2021September 8, 2022

Alexion Pharmaceuticals, which owns Soliris, one of the most expensive drugs on Earth, has agreed to sell AstraZeneca for $39 billion.

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

Danyelza: New Drug for Neuroblastoma That Can Cure It

by Julia Mardi and Mark Gubar April 16, 2021September 8, 2022

Naxitamab by Y-mAbs Therapeutics offers gorgeous treatment efficacy with unsafe adverse events.

Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Koselugo: First Drug to Treat Neurofibromatosis

by Tanya von Reuss April 14, 2021September 8, 2022

Selumetinib by AstraZeneca powerfully suppresses tumor growth in neurofibromatosis type 1.

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

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

by Tanya von Reuss, Mark Gubar, Alex Avisoni and Nik Resovski April 8, 2021September 8, 2022

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

heart
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Arcalyst: World’s First Drug to Treat Recurrent Pericarditis

by Tanya von Reuss March 30, 2021September 8, 2022

Rilonacept by Kiniksa Pharmaceuticals will reduce recurrent pericarditis to almost nothing.

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