[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…
[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…
[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…
[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…
[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…
[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…
[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…