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Daily Archives: July 12, 2021

Home » Archives for July 12, 2021
LYS-GM101 for GM1 Gangliosidosis Granted Fast Track Designation
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LYS-GM101 for GM1 Gangliosidosis Granted Fast Track Designation

  • Post author:Jessica Lynn
  • Post published:July 12, 2021
  • Post category:GM1 gangliosidosis

On July 8, 2021, gene therapy company Lysogene shared that its gene therapy candidate LYS-GM101, for patients with GM1 gangliosidosis, received Fast Track designation from the FDA. Currently, no treatments…

Continue Reading LYS-GM101 for GM1 Gangliosidosis Granted Fast Track Designation
Antibiotics Potentially Raise CRC Risk, Research Shows
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Antibiotics Potentially Raise CRC Risk, Research Shows

  • Post author:Jessica Lynn
  • Post published:July 12, 2021
  • Post category:Colorectal cancer

The ESMO World Congress on Gastrointestinal Cancer 2021 took place from June 30-July 3, 2021. During the Congress, researchers discussed data and research in the gastrointestinal field. According to MedScape,…

Continue Reading Antibiotics Potentially Raise CRC Risk, Research Shows
Study of the Week: Could This Discovery Help Drugs Cross the Blood-Brain-Barrier?
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Study of the Week: Could This Discovery Help Drugs Cross the Blood-Brain-Barrier?

  • Post author:James Moore
  • Post published:July 12, 2021
  • Post category:Rare Disease

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

Continue Reading Study of the Week: Could This Discovery Help Drugs Cross the Blood-Brain-Barrier?
Upadacitinib Meets All Phase 3 UC Trial Endpoints
source

Upadacitinib Meets All Phase 3 UC Trial Endpoints

  • Post author:Jessica Lynn
  • Post published:July 12, 2021
  • Post category:ulcerative Colitis

Towards the end of June 2021, biopharmaceutical company AbbVie shared that Upadacitinib (RINVOQ) reached both its primary and all secondary endpoints in a Phase 3 maintenance study evaluating the therapy…

Continue Reading Upadacitinib Meets All Phase 3 UC Trial Endpoints
Passion with a Purpose: How ANGEL AID CARES Uplifts Rare Mothers
ANGEL AID CARES

Passion with a Purpose: How ANGEL AID CARES Uplifts Rare Mothers

  • Post author:Jessica Lynn
  • Post published:July 12, 2021
  • Post category:Hunter Syndrome/MPS II (Hunter Syndrome)/Rare Disease

ANGEL AID CARES began as the brainchild of Cristol Barrett O’Loughlin, out of a shared desire to pay tribute to her own family experience and to uplift others in need.…

Continue Reading Passion with a Purpose: How ANGEL AID CARES Uplifts Rare Mothers
Approval Decision Deferred for Anal Cancer Drug Retifanlimab
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Approval Decision Deferred for Anal Cancer Drug Retifanlimab

  • Post author:Jessica Lynn
  • Post published:July 12, 2021
  • Post category:Anal Cancer

Thus far, retifanlimab, a drug designed to treat patients with anal cancer, has received Orphan Drug designation and Priority Review status. However, the approval process has not been entirely easy.…

Continue Reading Approval Decision Deferred for Anal Cancer Drug Retifanlimab
POTS from 100 to Zero in 2.3 seconds
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POTS from 100 to Zero in 2.3 seconds

  • Post author:katewild13@yahoo.com
  • Post published:July 12, 2021
  • Post category:POTS

Ever since I could remember I have had an aptitude for adventure. I like to be on the move, engage in sports and any out door activity and most importantly…

Continue Reading POTS from 100 to Zero in 2.3 seconds
Could Hepatocellular Carcinoma Cause One Million Deaths Annually by 2030?
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Could Hepatocellular Carcinoma Cause One Million Deaths Annually by 2030?

  • Post author:Rose Duesterwald
  • Post published:July 12, 2021
  • Post category:Hepatocellular Carcinoma

  Thanks to advances in immunotherapy, liver cancer (Hepatocellular carcinoma-HCC) has seen a rise in treatment options for patients in advanced stages of the disease. HCC is one of the…

Continue Reading Could Hepatocellular Carcinoma Cause One Million Deaths Annually by 2030?
This Cell Organelle Could be a Key Factor in Future Treatments
source: pixabay.com

This Cell Organelle Could be a Key Factor in Future Treatments

  • Post author:Rose Duesterwald
  • Post published:July 12, 2021
  • Post category:Rare Disease

   The answer lies in the loss of endoplasmic reticulum (ER) calcium and protein which leads to cardiovascular, musculoskeletal, viral, and neurodegenerative diseases. ER performs many important cellular functions including…

Continue Reading This Cell Organelle Could be a Key Factor in Future Treatments

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Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)

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Welcome to Study of the Week. We go in-depth and select a study we think is of particular interest, discussing details, explaining its importance, who may be impacted and lots more!

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