Healio21h
FDA approves Evrysdi tablets for spinal muscular atrophy
The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...
From liquid to solid: New technique uncovers disease-related changes in tiny droplets within our cells
Understanding the behavior of tiny droplets in our cells could aid the search for new treatments. A team of Leiden ...
Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update
Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...
Ultragenyx reports Q4 EPS ($1.39), consensus ($1.27)
Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...
Inability of cells to recycle fats can spell disease: Study reveals how a protein helps transport fats out of lysosomes
Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National ...
Tax Guru2d
Import Duty & GST Exemptions on Life-Saving Drugs for Rare Diseases
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 ...