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Editor’s Choice: Studies on Studies on Studies

Editor’s Choice: Studies on Studies on Studies

We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…

Continue Reading Editor’s Choice: Studies on Studies on Studies
In Canada, Medical Care is Covered. Now How Do You Afford Travel and Accommodations?

In Canada, Medical Care is Covered. Now How Do You Afford Travel and Accommodations?

In the US, medical costs come in the form of drug prescriptions, doctors visits, expensive surgeries, and visits to specialists. As originally reported in The Tyee, in Canada, patients are…

Continue Reading In Canada, Medical Care is Covered. Now How Do You Afford Travel and Accommodations?
Enrollment Finishes Early for a Late Stage Hemophilia B Gene Therapy Clinical Trial

Enrollment Finishes Early for a Late Stage Hemophilia B Gene Therapy Clinical Trial

According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…

Continue Reading Enrollment Finishes Early for a Late Stage Hemophilia B Gene Therapy Clinical Trial
Biohacking: A Small Group of Amateur Biologists Are Hoping to Market Their Knockoff of a One Million Dollar Gene Therapy

Biohacking: A Small Group of Amateur Biologists Are Hoping to Market Their Knockoff of a One Million Dollar Gene Therapy

  According to a recent article in Archive Today, an international band of amateur biologists, or "biohackers" announced that they intend to eventually market a knock-off of Glybera, a one…

Continue Reading Biohacking: A Small Group of Amateur Biologists Are Hoping to Market Their Knockoff of a One Million Dollar Gene Therapy
FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug

FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug

According to a press release from Maryland-based Viela Bio, the US Food and Drug Administration (FDA) recently agreed to review the Company's Biologics License Application for its neuromyelitis optica spectrum…

Continue Reading FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug
How Automated Text Messages Could Prevent Colon Cancer

How Automated Text Messages Could Prevent Colon Cancer

As reported in Forbes Magazine, Researchers at the University of Pennsylvania are exploring a new tool for encouraging patients to comply with health care recommendations for colonoscopies, an invasive procedure…

Continue Reading How Automated Text Messages Could Prevent Colon Cancer
The Results of This Phase 3 PKAN Clinical Trial are a Major Letdown

The Results of This Phase 3 PKAN Clinical Trial are a Major Letdown

According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently released the topline results from its phase 3 clinical trial. This clinical trial was testing the company's drug…

Continue Reading The Results of This Phase 3 PKAN Clinical Trial are a Major Letdown
Mystery: A Paralysis That Occurs in Children Every-Other-Year

Mystery: A Paralysis That Occurs in Children Every-Other-Year

  The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports…

Continue Reading Mystery: A Paralysis That Occurs in Children Every-Other-Year
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Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
*cancer that cannot be removed with surgery or has spread to other parts of the body. GEJ= gastroesophageal junction (where the esophagus joins the stomach)
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