Naxitamab by Y-mAbs Therapeutics offers gorgeous treatment efficacy with unsafe adverse events.
Rare/Orphan Diseases
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Koselugo: First Drug to Treat Neurofibromatosis
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
Novartis revealed the most expensive drug in the world. And it is amazing.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Arcalyst: World’s First Drug to Treat Recurrent Pericarditis
Rilonacept by Kiniksa Pharmaceuticals will reduce recurrent pericarditis to almost nothing.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Zynteglo: Gene Therapy for Beta-thalassemia
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
