Happy start-of-summer everyone! We're kicking off the summer with a wrap up on rare disease news this week. We have an article about a systemic mastocytosis study, and a clinical…
According to a story from The Spectator, Madeline Pitts, who has two forms of terminal cancer, will have June 9th be dubbed "Maddie Day" in her honor. She is thirteen…
According to a story from apnews.com, Mike Coble, a senior at Byron High School in southeastern Minnesota, was recently diagnosed with Guillain-Barre syndrome. Mike is on three different sports teams…
According to a story from tdn.com, the radio host Jamie Dupree will soon return to work after nearly a two year absence. However, he will not be doing any announcing…
According to Financial Buzz Chiasma, Inc. has reached a new clinical stage in its development of a treatment for acromegaly. The trial, known as CHIASMA OPTIMAL, tests a treatment currently…
On June 28th, 2018, the National Society for Phenylketonuria (NSPKU) is holding a special event that is meant to challenge Members of Parliament (MPs) across the UK. This challenge is…
Data from a Phase II clinical trial of the experimental treatment reldesemtiv for the treatment of spinal muscular atrophy has been announced. The drug has shown positive effects in the…
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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