According to a story from PR Newswire, Akcea Therapeutics, Inc. recently announced that the Portuguese Medicine Regulatory Authority (INFARMED) has announced its approval of reimbursement for inotersen (marketed as TEGSEDI®)…
In Spain, a young child showed signs of muscle atrophy and difficulty walking; she wasn't quite stable on her feet and struggled with balance and coordination. But it wasn't until…
Findings of the interim analysis from KEYNOTE-177 were presented at the virtual scientific meeting of the American Society of Clinical Oncology (ASCO) held in May 2020. The lead author…
By Danielle Bradshaw from In The Cloud Copy Expressive language sampling, or ELS, could potentially help researchers better measure how well treatments work for patients with fragile X syndrome or…
Arabel Standingbear sketches figures with lead pencils and paints realistic scenes with acrylics. Her poem was accepted by a museum. She has won in local art contests between schools, and…
Nicox SA, an international ophthalmology company, recently announced a Phase 3 clinical trial for NCX 470 to lower intraocular eye pressure. The article recently appeared in Eye Wire News. The…
Happy Friday! The world is full of difficult news right now. If you're looking for ways to educate yourself on some of the problems facing the world right now, especially…
An estimated 25-30 million Americans are part of the rare disease community. If you're one of them, you share a common (yet uncommon) experience: public scrutiny, difficulties in diagnoses, treatment…
Education, research, support, and patient advocacy: these are just four of the amazing things that rare disease or disorder foundations offer. Many foundations seek to offer programs and resources to…
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