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Daily Archives: March 12, 2021

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A World-first: Canadians Use Gene Therapy to Successfully Treat Fabry Disease

A World-first: Canadians Use Gene Therapy to Successfully Treat Fabry Disease

  • Post author:Rose Duesterwald
  • Post published:March 12, 2021
  • Post category:Fabry Disease

It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…

Continue Reading A World-first: Canadians Use Gene Therapy to Successfully Treat Fabry Disease
Nemvaleukin Alfa Granted Orphan Drug Status for Mucosal Melanoma
https://unsplash.com/photos/In4XVKhYaiI

Nemvaleukin Alfa Granted Orphan Drug Status for Mucosal Melanoma

  • Post author:Jessica Lynn
  • Post published:March 12, 2021
  • Post category:Melanoma

On March 11, 2021, global biopharmaceutical company Alkermes plc shared that its investigational drug candidate nemvaleukin alfa received Orphan Drug designation from the FDA. The treatment is designed for patients…

Continue Reading Nemvaleukin Alfa Granted Orphan Drug Status for Mucosal Melanoma
Charleston Family Spreads CSID Awareness After Daughter’s Diagnosis
source: pixabay.com

Charleston Family Spreads CSID Awareness After Daughter’s Diagnosis

  • Post author:Jessica Lynn
  • Post published:March 12, 2021
  • Post category:Congenital Sucrase-Isomaltase Deficiency

Altogether, there are over 7,000 rare diseases. Worldwide, 1 in 20 people live with a rare disease. But the journey to diagnosis can sometimes be confusing or difficult. So when…

Continue Reading Charleston Family Spreads CSID Awareness After Daughter’s Diagnosis
Dad Living with Dercum’s Disease and Leukemia Contracts COVID-19 Twice

Dad Living with Dercum’s Disease and Leukemia Contracts COVID-19 Twice

  • Post author:Kendall Mason
  • Post published:March 12, 2021
  • Post category:COVID-19/Dercum Disease/Leukemia

Living with a rare disease can be difficult and scary for some, especially during a global pandemic that has affected millions. This Nottingham father knows these feelings firsthand; he lives…

Continue Reading Dad Living with Dercum’s Disease and Leukemia Contracts COVID-19 Twice
Study Identifies New Alport Syndrome Mutations
source: pixabay.com

Study Identifies New Alport Syndrome Mutations

  • Post author:James Moore
  • Post published:March 12, 2021
  • Post category:Alport syndrome

According to a study from Hindawi, a team of scientists have reported the discovery of never before seen mutations that are linked to Alport syndrome, a rare kidney disease. These…

Continue Reading Study Identifies New Alport Syndrome Mutations
March is Cerebral Palsy Awareness Month
source: pixabay.com

March is Cerebral Palsy Awareness Month

  • Post author:James Moore
  • Post published:March 12, 2021
  • Post category:Cerebral Palsy/Cerebral palsy, spastic diplegic

The month of March is recognized as Cerebral Palsy Awareness Month, a time that is dedicated to spreading awareness among the general public and the medical community about cerebral palsy,…

Continue Reading March is Cerebral Palsy Awareness Month
Rare Classroom: Hemophilia
source: shutterstock

Rare Classroom: Hemophilia

  • Post author:James Moore
  • Post published:March 12, 2021
  • Post category:Hemophilia/Hemophilia A/Hemophilia B

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

Continue Reading Rare Classroom: Hemophilia
Neihulizumab for GvHD Receives Fast Track Designation
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Neihulizumab for GvHD Receives Fast Track Designation

  • Post author:Jessica Lynn
  • Post published:March 12, 2021
  • Post category:Graft versus Host Disease/Graft-versus-Host Disease

In a recent press release, biotech company AltruBio Inc. shared that its therapy neihulizumab (AbGn-168H) received Fast Track designation for the treatment of patients with steroid refractory acute graft-versus-host disease…

Continue Reading Neihulizumab for GvHD Receives Fast Track Designation
You Are Not Alone: Empowering the Advanced Kidney Cancer Community
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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