As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…
In early June 2021, Epidermolysis Bullosa News reported that Filsuvez (Oleogel-S10), a topical gel for epidermolysis bullosa (EB), received Priority Review designation from the FDA. Filsuvez Developed by Amryt Pharma,…
The Rare Disease Legislative Advocates (RDLA) webinar for the month of June took place on June 17, 2021. This month's webinar provided updates on a variety of legislative topics that…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
In a news release from June 23, 2021, Revolo Biotherapeutics ("Revolo") shared that the FDA approved an Investigational New Drug (IND) application for the company's '1104 peptide treatment. Altogether, '1104…
Mark Dalziel has big plans for this weekend; he's going to bike the length of Scotland within two days. He has a very good reason for doing so, which is…
Orphan Drug designation is a status granted to drugs or biologics intended to treat patients with rare conditions, defined as those affecting under 200,000 Americans. According to Pulmonary Hypertension News,…
Mal de débarquement, or "sickness of disembarkment," is a phenomenon which causes a bobbing, rocking, or swaying sensation, alongside other symptoms. With many people, mal de débarquement only lasts…
As reported in Charcot-Marie Tooth News; this year, Eurordis has kicked off a campaign to make sure those with rare diseases don’t get left behind in the coming century. Small…
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