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Daily Archives: January 8, 2018

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U.K. Gets Approval For $700,000 SCID Gene Therapy Treatment
Source: Pixabay

U.K. Gets Approval For $700,000 SCID Gene Therapy Treatment

  • Post author:Meagan Fulps
  • Post published:January 8, 2018
  • Post category:Primary Immunodeficiencies/SCID

'Tis the season for gene therapy approvals. Reported by Bloomberg, the U.K. just approved its most expensive medication to date. The treatment is for severe combine immune deficiency, or more…

Continue Reading U.K. Gets Approval For $700,000 SCID Gene Therapy Treatment
Woulda, Shoulda, Coulda for Rare Disease Families
Source: Pixabay

Woulda, Shoulda, Coulda for Rare Disease Families

  • Post author:Denise Crompton
  • Post published:January 8, 2018
  • Post category:Mucolipidosis Type III/Rare Disease

Pondering “If only” and “What if “ adds anxiety for rare disease families. It seems to be a part of human nature that we do a lot of looking back,…

Continue Reading Woulda, Shoulda, Coulda for Rare Disease Families
Potential Fabry Disease Treatment is Granted Orphan Drug Status
source: pixabay.com

Potential Fabry Disease Treatment is Granted Orphan Drug Status

  • Post author:James Moore
  • Post published:January 8, 2018
  • Post category:Fabry Disease/Rare Disease

The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed…

Continue Reading Potential Fabry Disease Treatment is Granted Orphan Drug Status
Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease
Source: Pixabay

Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease

  • Post author:Meagan Fulps
  • Post published:January 8, 2018
  • Post category:Rare Disease/RPE65-associated Leber Congenital Amaurosis

The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…

Continue Reading Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease
This Opera-Lover with PAH Got an Encore for Life
Source: pixabay.com

This Opera-Lover with PAH Got an Encore for Life

  • Post author:Sarah Lau
  • Post published:January 8, 2018
  • Post category:pulmonary arterial hypertension/Rare Disease

Have you been told that you had to stop doing your passion in life because of your rare disease? This is the story of opera lover, Charity Tillemann-Dick. Her life…

Continue Reading This Opera-Lover with PAH Got an Encore for Life
Thalassemia and Gaucher May Be Linked Suggests A Surprising New Case Study
Source: Pixabay

Thalassemia and Gaucher May Be Linked Suggests A Surprising New Case Study

  • Post author:Samuel Sachs
  • Post published:January 8, 2018
  • Post category:Beta Thalassemia/Gaucher Disease

A new case study suggests that the blood disorder, thalassemia, may be present in people with Gaucher disease. Both diagnosis and treatment become more complicated if this hypothesis proves true.…

Continue Reading Thalassemia and Gaucher May Be Linked Suggests A Surprising New Case Study
The FDA Just Fast Tracked A Potential New Treatment For Glioblastoma
PDPics / Pixabay

The FDA Just Fast Tracked A Potential New Treatment For Glioblastoma

  • Post author:James Moore
  • Post published:January 8, 2018
  • Post category:Glioblastoma/Rare Disease

DelMar Pharmaceuticals announced that its flagship cancer treatment drug, called VAL-083, was given "Fast Track" status by the Food and Drug Administration. VAL-083 is a possible treatment for a type…

Continue Reading The FDA Just Fast Tracked A Potential New Treatment For Glioblastoma
Supermom Cori Salchert Fosters Terminally Ill Children
Source: Pixabay

Supermom Cori Salchert Fosters Terminally Ill Children

  • Post author:Meagan Fulps
  • Post published:January 8, 2018
  • Post category:Hypoxic Ischemic Encephalopathy/Rare Disease

Cori Salchert is a hero. Recently featured on the Today Show, Cori was honored for being the ultimate supermom, taking in foster children who are sick with terminal diseases, report…

Continue Reading Supermom Cori Salchert Fosters Terminally Ill Children

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