Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
Prader-Willi syndrome (PWS) is a rare genetic disorder affecting approximately 1 in 10,000–30,000 individuals worldwide. It is the leading syndromic cause of obesity, with an estimated 400,000 cases globally and…
A Wake Forest University School of Medicine study reports that two existing medications (empagliflozin (Jardiance), a diabetes drug, and intranasal insulin) could improve brain health in individuals with mild cognitive…
BridgeBio Pharma has marked another significant milestone in rare disease research, announcing a second late-stage clinical trial success—this time with encaleret, a therapy for the uncommon endocrine disorder autosomal dominant…
As reported by drugs.com, at the American College of Rheumatology (ACR) Convergence congress, Novartis presented encouraging results from its Phase III NEPTUNUS-1 and NEPTUNUS-2 trials, highlighting the potential of ianalumab…
Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and…
Sanofi has announced encouraging results from its Phase 2 ElevAATe clinical trial evaluating efdoralprin alfa (SAR447537), a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the treatment of emphysema caused…
This patient story is sponsored by AVEO Pharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
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