Editor’s Choice: International Acromegaly Day

Happy Friday!

November 1 is International Acromegaly Day. For those unaware, Acromegaly is a rare condition characterized by the presence of an elevated level of growth hormone and typically effects people between the ages of 30 and 50. Our partners over at the World Alliance of Pituitary Organizations have more info here. If you’d like to help spread awareness of this condition or are looking to connect with other patients, well, you have options. Acromegaly Community USA, the Pituitary Foundation UK, Vancouver Acromegaly Support Group and Atlantic Acromegaly Support are all good places to start.

In this week’s Editor’s Choice, we have details about a new clinical trial evaluating an upcoming treatment for HAE. This new therapy for Duchenne Muscular Dystrophy was approved by the FDA this August and is nearing approval in Canada. And finally, here’s why a novel combo for Renal Cell Carcinoma was granted priority review status. We hope you have a great weekend!

 

New Results Published from Phase-3 Clinical Trial Evaluating Berotralstat for HAE

The data was published in the Journal of Allergy and Clinical Immunology.

 

New Therapy for Duchenne Muscular Dystrophy Recently Approved by FDA, Nears Approval in Canada

Researchers have turned to synthetic molecules that resemble DNA but are not actually DNA.

 

Opdivo-Cabometyx Combo for Renal Cell Carcinoma Given Priority Review Status

The FDA will make its decision on the combination by the end of February 2021.

 


Do you have a rare disease experience of your own? Share with us here.

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