The 2019 Global Genes Rare Patient Advocacy Summit was filled with incredible presentations that highlighted steps for progress in research and advocacy organization, provided solidarity for the rare community, and…
According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…
The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…
According to Newswise, new findings indicate that electrical stimulation of the spinal cord and brain may be effective in treating symptoms of a group of rare neurological movement disorders, neurodegenerative ataxias.…
According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…
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