Facebook-f Twitter Instagram Podcast Youtube Tiktok Linkedin-in Pinterest Envelope Share
SHARE YOUR STORY!
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet the Patient Worthy Team
    • Collaborative Content On Patient Worthy
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
    • Patient Stories
  • Resources
    • Library
    • Events
    • Patient Worthy FAQs
  • Podcast
  • Contact
Menu
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet the Patient Worthy Team
    • Collaborative Content On Patient Worthy
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
    • Patient Stories
  • Resources
    • Library
    • Events
    • Patient Worthy FAQs
  • Podcast
  • Contact
  • Join PW

1459 Search Results Found

You searched for: "study of the week"
  1. Home>
  2. Search results for “study of the week”>
  3. Page 135
Rare Epilepsy Updates:  One New Drug, Two Clinical Trials, and Orphan Drug Designation

Rare Epilepsy Updates:  One New Drug, Two Clinical Trials, and Orphan Drug Designation

  A recent article published in Globe Newswire heralded Marinus Pharmaceuticals’ new drug, ganaxolone. The drug has been studied in over 1600 patients and is now being tested in two clinical…

Continue Reading Rare Epilepsy Updates:  One New Drug, Two Clinical Trials, and Orphan Drug Designation
Is the FDA Approving Drugs too Quickly?

Is the FDA Approving Drugs too Quickly?

   At times there seems to be no happy medium in life. A recent article in Bloomberg claims that the speed at which the FDA is approving newly developed drugs is…

Continue Reading Is the FDA Approving Drugs too Quickly?
Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency

Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency

  Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…

Continue Reading Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency
Early Data Looks Encouraging for an Experimental Thalassemia Treatment

Early Data Looks Encouraging for an Experimental Thalassemia Treatment

According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…

Continue Reading Early Data Looks Encouraging for an Experimental Thalassemia Treatment
How Gene Therapy is Being Applied to Sickle Cell Disease

How Gene Therapy is Being Applied to Sickle Cell Disease

  Grajevis Bakatunkanda lived in the Democratic Republic of the Congo, where he received treatment for malaria after experiencing weekly pain crisis. The treatment he received did not work, as…

Continue Reading How Gene Therapy is Being Applied to Sickle Cell Disease
How Researchers Used Multiple Sclerosis to Understand Autoimmune Encephalitis

How Researchers Used Multiple Sclerosis to Understand Autoimmune Encephalitis

  Autoimmune encephalitis is a group of diseases that was only identified about a decade ago. The newness of this condition makes it difficult to completely understand it. Dr Robb…

Continue Reading How Researchers Used Multiple Sclerosis to Understand Autoimmune Encephalitis
Pierre Robin Sequence: EU Family Seeks Appeal After Denial of Treatment Coverage

Pierre Robin Sequence: EU Family Seeks Appeal After Denial of Treatment Coverage

Philippe Pakter’s daughter Lysiane was born on March 29th 2017 in Lyon, France. However, soon after her birth, Philippe and his partner Delphine were hit with shocking news: Lysiane was…

Continue Reading Pierre Robin Sequence: EU Family Seeks Appeal After Denial of Treatment Coverage
The Results From This Immune Thrombocytopenia Trial are Good News for Patients

The Results From This Immune Thrombocytopenia Trial are Good News for Patients

According to a story from ucb.com, the biopharmaceutical company UCB has recently released the final results from its phase 2 clinical trial which tested the company's investigational monoclonal antibody rozanolixizumab…

Continue Reading The Results From This Immune Thrombocytopenia Trial are Good News for Patients
  • Go to the previous page
  • 1
  • …
  • 132
  • 133
  • 134
  • 135
  • 136
  • 137
  • 138
  • …
  • 183
  • Go to the next page

Featured


Picture of Family


Metastatic Breast Cancer: Navigating Grief


Picture of Ralph Family walking


Rethinking What It Means to Live With Acromegaly


Illustration of mentor program members


The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

SHARE YOUR STORY!
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

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 Notice
Privacy Policy for CA Residents
EU/UK Privacy Notice
Data Privacy Framework: Consumer Privacy Policy
Consumer Health Data Privacy Policy
Cookie Notice

Facebook-f Twitter Instagram Podcast Youtube Tiktok Linkedin-in Pinterest Envelope

© Copyright 2024 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