by Stacey Hawkins | Nov 5, 2019 | Genetic Disease
A Breath of fresh air for cystic fibrosis patients It’s not often that a radical treatment for a chronic, often fatal, disease comes around. The WEEKLY takes notice when one does. Vertex Pharmaceuticals’ (Boston, MA) innovative new drug, Trikafta, promises better... 
by Stacey Hawkins | Jul 27, 2017 | Groundbreaking New Therapies in Development
PROPERLY FOLDING MISFOLDED DISEASE PROTEINS Amicus Therapeutics (Cranbury, NJ) found itself in the news earlier this month when the FDA agreed to review the company’s new drug application for their investigational therapy to treat Fabry’s disease. The drug under... 
by Stacey Hawkins | Jan 7, 2016 | Regulation
Treatment Options Broaden Driven by 19 new orphan approvals, a whopping 45 novel drugs were cleared for the marketplace in 2015. Additionally, new approvals opened up treatment options for Americans with diseases ranging from breast and lung cancers to irritable bowel... 
by Stacey Hawkins | Jul 9, 2015 | Uncategorized
Biotech Changes The Game The FDA approval of Vertex Pharmaceutical’s (Boston, MA) Orkambi last week further shifted the paradigm of treating cystic fibrosis (CF) for up to half of its sufferers—moving from a management of symptoms approach to targeting the underlying... 
by Stacey Hawkins | Feb 5, 2015 | Precision Medicine
During President Obama’s State of the Union address last month, a cystic fibrosis patient named Bill Elder sat beside First Lady Michelle Obama. Diagnosed with the disease at 8 years old, Mr. Elder is “healthier now than ever before” at age 27, thanks...
