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Daily Archives: April 30, 2021

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Editor’s Choice: Duchenne, GSD and Orphan Drugs

Editor’s Choice: Duchenne, GSD and Orphan Drugs

  • Post author:Colby Rogers
  • Post published:April 30, 2021
  • Post category:Duchenne Muscular Dystrophy/Glycogen Storage Disease Type I

Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen…

Continue Reading Editor’s Choice: Duchenne, GSD and Orphan Drugs
“The Boy with the Turquoise Ribbon” Helps Families Understand CDH
AnnieSpratt / Pixabay

“The Boy with the Turquoise Ribbon” Helps Families Understand CDH

  • Post author:Jessica Lynn
  • Post published:April 30, 2021
  • Post category:Congenital Diaphragmatic Hernia

Oftentimes, the rare disease journey can be difficult. Diagnosis may take years. Some conditions are still relatively unknown by doctors. Families must not only learn about these conditions, but how…

Continue Reading “The Boy with the Turquoise Ribbon” Helps Families Understand CDH
This Mother Used Social Media to Talk About Raynaud’s Syndrome

This Mother Used Social Media to Talk About Raynaud’s Syndrome

  • Post author:Kendall Mason
  • Post published:April 30, 2021
  • Post category:Rare Disease

Many Twitter users were confused and concerned when Julie O'Mahony posted a picture of her mother's hand with two of the fingers white and completely drained of color. However, while…

Continue Reading This Mother Used Social Media to Talk About Raynaud’s Syndrome
World Orphan Drug Congress: Rare Disease Trials and Patient Registries
source: pixabay.com

World Orphan Drug Congress: Rare Disease Trials and Patient Registries

  • Post author:James Moore
  • Post published:April 30, 2021
  • Post category:Ovarian Cancer/Polycystic Ovary Syndrome/Rare Disease

On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…

Continue Reading World Orphan Drug Congress: Rare Disease Trials and Patient Registries
Study Links Diplopia to Parkinson’s Disease
source: pixabay.com

Study Links Diplopia to Parkinson’s Disease

  • Post author:Jessica Lynn
  • Post published:April 30, 2021
  • Post category:Parkinson's Disease

Did you know that an estimated 30% of patients with Parkinson's disease (PD) experience diplopia (double vision)? According to Parkinson's News Today, a large longitudinal study determined that diplopia is…

Continue Reading Study Links Diplopia to Parkinson’s Disease
Fish Oil Supplements Could Increase AF Risk
source: pixabay.com

Fish Oil Supplements Could Increase AF Risk

  • Post author:Jessica Lynn
  • Post published:April 30, 2021
  • Post category:Atrial Fibrillation

For many people, fish oil supplements are part of their health routine. I know that I have a bottle sitting in my vitamin cabinet. These supplements offer omega-3 fatty acids…

Continue Reading Fish Oil Supplements Could Increase AF Risk
Brother with TSC Motivates Harvard-Bound Sister
source: pixabay.com

Brother with TSC Motivates Harvard-Bound Sister

  • Post author:Kendall Mason
  • Post published:April 30, 2021
  • Post category:Tuberous Sclerosis/Tuberous Sclerosis Complex

Chiprez Ramirez describes her younger brother Francisco as her "little ray of sunshine." Talking to MSN, she said that his rare disease journey and strength are what motivate her to…

Continue Reading Brother with TSC Motivates Harvard-Bound Sister
Rare Classroom: Glycogen Storage Disease Type IB
source: shutterstock

Rare Classroom: Glycogen Storage Disease Type IB

  • Post author:James Moore
  • Post published:April 30, 2021
  • Post category:Glycogen Storage Disease Type I

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: Glycogen Storage Disease Type IB
Debbie Allen Is Helping to Educate About Chronic Kidney Disease
source: pixabay.com

Debbie Allen Is Helping to Educate About Chronic Kidney Disease

  • Post author:Kendall Mason
  • Post published:April 30, 2021
  • Post category:Chronic Kidney Disease

The National Kidney Foundation has partnered with Bayer to launch and run the "Are You the 33%?" campaign, which intends to raise awareness about the heightened risk of chronic kidney…

Continue Reading Debbie Allen Is Helping to Educate About Chronic Kidney Disease
TRS02 Pre-IND Meeting Successfully Completed
Aymanjed / Pixabay

TRS02 Pre-IND Meeting Successfully Completed

  • Post author:Jessica Lynn
  • Post published:April 30, 2021
  • Post category:Diabetic macular edema/Glaucoma/Panuveitis/Uveitis

In a recent news release, pharmaceutical company Tarsier Pharma ("Tarsier") shared that it held a successful pre-Investigational New Drug (pre-IND) meeting with the FDA. The meeting focused on TRS02, a…

Continue Reading TRS02 Pre-IND Meeting Successfully Completed

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