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News of the Week

Summary of industry news related to rare diseases and genetic diagnostics industry
33 posts
News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

News of the Week

[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…

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