[May 19, 2022] News of the Week
Rare disease pharmaceutical industry trendsRocket Pharmaceuticals, Inc. announced the progress of phase 2 clinical trials for Fanconi Anemia treatment and Phase 1 clinical trials for Danaon…
[May 12, 2022] News of the Week
Rare disease pharmaceutical industry trendsThe US Food and Drug Administration's Center for Drug Evaluation and Research (CDER) has announced the Accelerating Rare Disease Cures (ARC) program…
[May 6, 2022] News of the Week
Rare disease pharmaceutical industry trendsThe FDA approved the first treatment for penicillamine-tolerant patients with Wilson's disease. Wilson's disease is a rare genetic disorder in which copper…
[April 28, 2022] News of the Week
Rare disease pharmaceutical industry trendsThe Czech health minister has urged the EU to jointly purchase medicines for rare diseases to lower the price of drugs and…
[April 21, 2022] News of the Week
Rare disease pharmaceutical industry trendsU.S FDA designated Aytu BioPharma’s Vascular Ehlers Danlos Syndrome treatment ‘AR101’ as a fast track. The treatment had already been…
[April 14, 2022] News of the Week
Rare disease pharmaceutical industry trendsTakeda and Evozyne signed a $400 million contract to develop gene therapy for rare diseases. If Evozyne's AI and machine learning technology…
[April 7, 2022] News of the Week
Rare disease diagnostics & researchResearchers from the Genomic Answers for Kids (GA4K) project have published a study showing that long-read sequencing technology and machine learning technology…
[March 31, 2022] News of the Week
Rare disease pharmaceutical industry trendsSanofi's first treatment for acid sphingomyelinase deficiency (ASMD), olipudase alfa (Xenpozyme), was approved in Japan. It is expected to be approved in…
[March 24, 2022] News of the Week
Rare disease pharmaceutical industry trendsA treatment for CDKL-5 deficiency (CDD), a rare genetic disorder, has been approved by the FDA. This makes Ztalmy, developed by Marinus…
[March 17, 2022] News of the Week
Rare disease pharmaceutical industry trendsIn collaboration with Scenic Biotech, Roche plans to conduct clinical trials for three rare disease treatments. The three drugs are treatments for…
[March 11, 2022] News of the Week
Rare disease pharmaceutical industry trendsNovartis plans to develop more central nervous system-targeted therapies through its deal with Voyager Therapeutics. With Voyager's drug delivery technology, Novartis seeks…
[March 3, 2022] News of the Week
Rare disease pharmaceutical industry trendsDissemination of treatments for rare diseases has many challenges even after development and marketing approval. Among them, this article deals with the…