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 ...

Network attached storage is the most versatile way to store data, but that's just one of the many benefits of buying a NAS device. We've tested the top models: See which will work best for your ...

Diseases of the nervous system refer to disorders that affect any part of the central nervous system (brain, spinal cord or cranial nerves) or peripheral nervous system. During the pathogenesis of ...

High blood pressure affects multiple organs. Not only can hypertension add stress to the heart and damage arteries, leading to heart disease, but it also can cause strokes and kidney disease as ...