Top 5 Rare Disease Articles of the Week

RDR Staff

Was a busy week at the FDA with 3 drugs getting approved for new rare disease indications. In addition, our top 5 rare disease articles included our 10-minute video of the many celebrities and rare disease KOLs that walked the blue carpet at Global Genes’ Tribute to Champions of Hope.

Global Genes Blue Carpet

Hanging out with a few friends at Global Genes - Ed O'Neill, Audrey Nethery, Sam Kimera, Alex Kimera, Taylor Hicks, Cali Tucker, DJ Ravi,Travis Flores, Travis Lively, Nancy Harris, Phillip Reilly, Illna Gozes, Arthur Landers, Elif Oral, Daniel Ory, Madison McLaughlin, Geoff Reeves, Sick Chicks, Brooke Lewis, and many many more.
Click here to see the complete video.

FDA Approves Biosimilar Amjevita to Treat Crohn's Disease and Other Inflammatory Diseases

The FDA has approved Amgen’s Amjevita, a biosimilar to adalimumab for the treatment of Crohn’s disease and 6 other inflammatory diseases. 

Amjevita was approved based on analytical, nonclinical, pharmacokinetic and clinical data supporting biosimilarity to adalimumab.
Click here to read the complete article.

Does Michael Phelps Have Marfan Syndrome?

What sets Michael apart from other swimmers is his physical traits. He is extremely tall with a thin physique, a protruding chin and disproportionately long arms. Great attributes to be an Olympic swimmer.

These physical attributes are also associated with the rare disease, Marfan’s syndrome. 
Click here to read the complete article.

FDA Approves Stelara for Treatment of Adult Crohn's Disease

Along with approving Amjevita, a biosimilar to adalimumab, the FDA has also just given the green light to Janssen Biotech’s Stelara (ustekinumab) for the treatment of moderately to severely active Crohn’s disease in patients 18 years or older.
Click here to read the complete article.

FDA Approves Vertex's Orkambi for Cystic Fibrosis Children 6-11 Years Old

The FDA has approved Vertex Pharmaceuticals’ drug Orkambi (lumacaftor/ivacaftor) for use in children ages 6-11 years old with cystic fibrosis (CF) who have 2 copies of the F508del mutation.
Click here to read the complete article.

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