The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...

Understanding the behavior of tiny droplets in our cells could aid the search for new treatments. A team of Leiden ...

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National ...

Government of India has provided tax relief on life-saving drugs and medicines used for rare diseases. As per the Ministry of Finance’s response in Rajya Sabha on February 11, 2025, basic customs duty ...

Orchard Therapeutics, a Kyowa Kirin company, today announced presentations from seven abstracts from across its hematopoietic stem cell (HSC) gene therapy portfolio will be featured at the 21st Annual ...

We may receive compensation when you click on links to products we review. Please view our affiliate disclosure. Cloud storage has become an essential tool for securely storing, accessing, and sharing ...

But this data can be saved if you have secure backups. One such way to create reliable backups is by using cloud storage that doesn't rely on external devices. We’ve rounded up the best cloud ...

One of the easiest ways to get your files stored online is by using cloud storage, and the best way to start your journey into the cloud is with free cloud storage provider, or a free cloud ...