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News of the Week

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

[June 30, 2022] News of the Week

Rare disease pharmaceutical industry trendsTwo cases of Sanofi's rare disease enzyme replacement therapy (ERT) have been approved by the European Commission. One is Xenpoxyme, a treatment…

News of the Week

[June 23, 2022] News of the Week

Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…

News of the Week

[June 16, 2022] News of the Week

Rare disease pharmaceutical industry trends In the EU, the need for improvement of related policies is being emphasized for the early diagnosis of rare diseases and…

News of the Week

[June 9, 2022] News of the Week

Rare disease pharmaceutical industry trendsUCLA researchers have published an animal study showing that guanidinoacetate methyltransferase deficiency (GAMT) can be treated through gene therapy. The researchers transferred…

News of the Week

[June 3, 2022] News of the Week

Rare disease pharmaceutical industry trendsThe FDA has approved Roche's SMA (Spinal Muscular Atrophy) treatment Evrysdi for use in infants under 2 months of age. This makes…

News of the Week

[May 26, 2022] News of the Week

Rare disease pharmaceutical industry trends The rare disease pharmaceutical market has been growing rapidly since the Orphan Drug Act in 1983. This article introduces the rare…

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…

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