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spinal muscular atrophy (SMA)

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.

Alltrna.
Posted inMetabolism, Neuroscience, Oncology, Pharma & Biotech, Rare/Orphan Diseases

Alltrna: World’s First Drugs Based on Transfer RNAs

November 9, 2021September 8, 2022

A completely new therapeutic modality will help treat both rare and common diseases.

Zolgensma: Gene Therapy That Cure Spinal Muscular Atrophy. The Most Comprehensive Review
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

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

April 8, 2021September 8, 2022

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

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