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Daily Archives: December 26, 2018

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Experimental Fabry Disease Treatment Gets Orphan Drug Designation
geralt / Pixabay

Experimental Fabry Disease Treatment Gets Orphan Drug Designation

  • Post author:James Moore
  • Post published:December 26, 2018
  • Post category:Fabry Disease

According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…

Continue Reading Experimental Fabry Disease Treatment Gets Orphan Drug Designation
FDA Green Lights Drugs for Two Rare Diseases  

FDA Green Lights Drugs for Two Rare Diseases  

  • Post author:Jean Martell
  • Post published:December 26, 2018
  • Post category:Blastic plasmacytoid dendritic cell neoplasm/paroxysmal nocturnal hemoglobinuria

Big news out of the FDA last week! Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval…

Continue Reading FDA Green Lights Drugs for Two Rare Diseases  
Cancer Drug KEYTRUDA Approved for Merkel Cell Carcinoma by the FDA
analogicus / Pixabay

Cancer Drug KEYTRUDA Approved for Merkel Cell Carcinoma by the FDA

  • Post author:James Moore
  • Post published:December 26, 2018
  • Post category:Merkel cell cancer/Rare Disease

According to a story from BioSpace, the biopharmaceutical company Merck recently announced that the US Food and Drug Administration (FDA) has approved the company's cancer drug KEYTRUDA in a new…

Continue Reading Cancer Drug KEYTRUDA Approved for Merkel Cell Carcinoma by the FDA
Ulcerative Colitis Linked to Increased Risk of Prostate Cancer
derneuemann / Pixabay

Ulcerative Colitis Linked to Increased Risk of Prostate Cancer

  • Post author:James Moore
  • Post published:December 26, 2018
  • Post category:Rare Disease/ulcerative Colitis

According to a story from WebMD, a recent study revealed that men with Crohn's disease, ulcerative colitis, or other forms of inflammatory bowel disease are at an increased risk of…

Continue Reading Ulcerative Colitis Linked to Increased Risk of Prostate Cancer
After Dealing With Chronic Fatigue Syndrome for Years, This Patient is Giving Back
Sweetaholic / Pixabay

After Dealing With Chronic Fatigue Syndrome for Years, This Patient is Giving Back

  • Post author:James Moore
  • Post published:December 26, 2018
  • Post category:Chronic Fatigue Syndrome

According to a story from gazettelive.co.uk, Jenni Mawson, who is currently 19 years old, has faced a years long struggle with chronic fatigue syndrome, which is also known as myalgic…

Continue Reading After Dealing With Chronic Fatigue Syndrome for Years, This Patient is Giving Back
New Drug Could Improve Sleep for Smith-Magenis Syndrome Patients!

New Drug Could Improve Sleep for Smith-Magenis Syndrome Patients!

  • Post author:Trudy Horsting
  • Post published:December 26, 2018
  • Post category:Non-24/Smith-Magenis Syndrome

Smith-Magenis Syndrome (SMS) is caused by a deletion of the 17p 2,3  chromosome. the condition affects 1 in every 15,000-25,000 individuals. It often results in a sleep disorder, and various mental, physical,…

Continue Reading New Drug Could Improve Sleep for Smith-Magenis Syndrome Patients!
Study Refutes Claims That Orphan Drugs Contribute to Rising Prices and Costs
jarmoluk / Pixabay

Study Refutes Claims That Orphan Drugs Contribute to Rising Prices and Costs

  • Post author:James Moore
  • Post published:December 26, 2018
  • Post category:Rare Disease

According to a story from BioSpace, a report on the results of the Orphan Drug Act of 1983 that was originally commissioned by the National Organization for Rare Disorders (NORD)…

Continue Reading Study Refutes Claims That Orphan Drugs Contribute to Rising Prices and Costs
5 Things My Boyfriend Has Had to Get Used to Since Dating a Spoonie

5 Things My Boyfriend Has Had to Get Used to Since Dating a Spoonie

  • Post author:Patient Worthy Contributor
  • Post published:December 26, 2018
  • Post category:Dysautonomia/Lyme Disease/POTS

As much as there is so much to talk about regarding dating while battling a chronic illness, I want to focus for a moment on what my long-term boyfriend has…

Continue Reading 5 Things My Boyfriend Has Had to Get Used to Since Dating a Spoonie
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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