• Rare Disease News
  • contribute@patientworthy.com
Facebook-f Instagram Linkedin-in Pinterest Twitter
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet Our Team
    • Collaborative Content On Patient Worthy
    • Contact Us
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
    • Patient Stories
  • Resources
    • Library
    • Memes
    • Events
    • Patient Worthy FAQs
  • Listen
  • Languages
    • Spanish / Español
    • Russian / русский
    • French / Français
  • Log In
Menu
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet Our Team
    • Collaborative Content On Patient Worthy
    • Contact Us
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
    • Patient Stories
  • Resources
    • Library
    • Memes
    • Events
    • Patient Worthy FAQs
  • Listen
  • Languages
    • Spanish / Español
    • Russian / русский
    • French / Français
  • Log In
  • Join PW

Daily Archives: June 21, 2022

Home » Archives for June 21, 2022
Justin Bieber’s Journey with Ramsay Hunt Syndrome

Justin Bieber’s Journey with Ramsay Hunt Syndrome

  • Post author:Alyssa Stevens
  • Post published:June 21, 2022
  • Post category:Ramsay Hunt syndrome

Popular Canadian singer Justin Bieber stunned the world when he announced he was diagnosed with Ramsay Hunt syndrome following his facial paralysis. Ramsay Hunt Syndrome Ramsay Hunt syndrome (herpes zoster…

Continue Reading Justin Bieber’s Journey with Ramsay Hunt Syndrome
Canada Approves its Third Drug For Amyotrophic Lateral Sclerosis (ALS)

Canada Approves its Third Drug For Amyotrophic Lateral Sclerosis (ALS)

  • Post author:Rose Duesterwald
  • Post published:June 21, 2022
  • Post category:Amyotrophic Lateral Sclerosis

Thanks to Amylyx Pharmaceuticals, Canada becomes the first country worldwide to have access to an ALS drug branded Albrioza (originally AMX0035). According to an article in Yahoo News, the drug…

Continue Reading Canada Approves its Third Drug For Amyotrophic Lateral Sclerosis (ALS)
Ilixadencel for GISTs Granted Orphan Drug Designation
https://unsplash.com/photos/tV-RX0beDp8

Ilixadencel for GISTs Granted Orphan Drug Designation

  • Post author:Jessica Lynn
  • Post published:June 21, 2022
  • Post category:Gastrointestinal Stromal Tumors

In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Do you know what makes a…

Continue Reading Ilixadencel for GISTs Granted Orphan Drug Designation
Patient Story: Meghan O’Rourke Talks Chronic Illnesses in Her New Novel
Pexels / Pixabay

Patient Story: Meghan O’Rourke Talks Chronic Illnesses in Her New Novel

  • Post author:Alyssa Stevens
  • Post published:June 21, 2022
  • Post category:Rare Disease

Meghan Rourke saw the need for more open conversations about chronic illnesses, sparking her to write The Invisible Kingdom: Reimaging Chronic Illness. In an excerpt from her novel, she shares…

Continue Reading Patient Story: Meghan O’Rourke Talks Chronic Illnesses in Her New Novel
Study Update: AT-01 for Systemic Amyloidosis

Study Update: AT-01 for Systemic Amyloidosis

  • Post author:Kendall Mason
  • Post published:June 21, 2022
  • Post category:Amyloidosis

From June 11th until the 14th, the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting was held in Vancouver, Canada. At this meeting, Attralus Inc. shared new data…

Continue Reading Study Update: AT-01 for Systemic Amyloidosis
Zolgensma Safe in Children with SMA Heavier than Clinical Trial Weights
cherylholt / Pixabay

Zolgensma Safe in Children with SMA Heavier than Clinical Trial Weights

  • Post author:Jessica Lynn
  • Post published:June 21, 2022
  • Post category:Spinal Muscular Atrophy

In clinical trials evaluating Zolgensma for children with spinal muscular atrophy (SMA), the maximum weight of participants was 18.7 pounds. However, parents with children weighing over that weight might be…

Continue Reading Zolgensma Safe in Children with SMA Heavier than Clinical Trial Weights
A New Study Finds That Gene Therapy May Prevent or Reverse Pitt-Hopkins Syndrome

A New Study Finds That Gene Therapy May Prevent or Reverse Pitt-Hopkins Syndrome

  • Post author:Rose Duesterwald
  • Post published:June 21, 2022
  • Post category:Pitt-Hopkins Syndrome

In normal development, genes are usually inherited in pairs - with one copy from the father and one inherited from the mother. When one copy of the TCF4 gene, which…

Continue Reading A New Study Finds That Gene Therapy May Prevent or Reverse Pitt-Hopkins Syndrome

OF INTEREST



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

GET INVOLVED

Make a difference, share your experiences and get paid. Opt-in & join Patient Worthy’s panel for paid opportunities such as Surveys, Market Research, Patient Advisory Panels & much more.

JOIN NOW

STUDY OF THE WEEK

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!

READ MORE

RARE CLASSROOM

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 mysterious diseases and conditions. This series is an opportunity to learn the basics about some of the diseases that almost no one hears much about.

READ MORE
SIGN UP FOR OUR NEWSLETTER
We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.
Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!

Let’s Work Together!

Partner With Us
Submit a Story
Become a Contributor

Keep Up to Date

Subscribe to Our Newsletter
Check Out Rare Events
Get Inspired By Our Memes

Learn More

About Us
Rare Diseases and Conditions
Terms of Use
Privacy Policy
Privacy Policy for CA Residents
EU Privacy Notice

© Copyright Patient Worthy

Sign Up With a Patient Worthy Account and Share Your Rare Story

- OR -

Sign Up For Our Patient Panel

Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.

More Info

We're Happy You're Here!

What best describes you when it comes to rare disease? (check all that apply)

What rare disease(s)/conditions are most important to you?

Visit Home Page or

Thank you for signing up for a Patient Worthy Account!

Have a rare disease story to share? Let us know

Share Story

- OR -

Sign Up For Our Patient Panel

Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.

More Info