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Daily Archives: December 27, 2021

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Study Launches to Evaluate AT-007 for SORD Deficiency
source: pixabay.com

Study Launches to Evaluate AT-007 for SORD Deficiency

  • Post author:Jessica Lynn
  • Post published:December 27, 2021
  • Post category:Rare Disease

According to a December 16, 2021 news release from biopharmaceutical company Applied Therapeutics, Inc., the company has initiated a registrational Phase 2/3 INSPIRE clinical trial to evaluate AT-007 for patients…

Continue Reading Study Launches to Evaluate AT-007 for SORD Deficiency
Holidays with a Chronic Illness

Holidays with a Chronic Illness

  • Post author:Patient Worthy Contributor
  • Post published:December 27, 2021
  • Post category:Rare Disease

Thinking about the year-end holidays brings up a kaleidoscope of images, and an array of emotions for me. And everyone else too, I imagine. There are the vivid memories of…

Continue Reading Holidays with a Chronic Illness
GM1 Gangliosidosis Gene Therapy Shows Promise In Early Trial So Far
source: shutterstock.com

GM1 Gangliosidosis Gene Therapy Shows Promise In Early Trial So Far

  • Post author:James Moore
  • Post published:December 27, 2021
  • Post category:GM1 gangliosidosis

According to a story from globenewswire.com, the gene therapy company Sio Gene Therapies, Inc., has recently released encouraging interim findings from its phase 1/2 clinical trial. This trial is evaluating…

Continue Reading GM1 Gangliosidosis Gene Therapy Shows Promise In Early Trial So Far
Christmas in the Hospital: Gareth’s Story
Source: Pixabay

Christmas in the Hospital: Gareth’s Story

  • Post author:Kendall Mason
  • Post published:December 27, 2021
  • Post category:Anaplastic large cell lymphoma/Graft versus Host Disease

  Gareth Crabb is a thoughtful and caring six-year-old boy from Swansea, U.K. He is spending his second Christmas in the hospital this year, as he has anaplastic large cell…

Continue Reading Christmas in the Hospital: Gareth’s Story

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