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Vincenzo’s Journey with Glanzmann Thrombasthenia

Vincenzo’s Journey with Glanzmann Thrombasthenia

Editor's Note: Patient Worthy is proud to bring you this story from our friends at the Glanzmann Research Foundation. To see the article in its original format, please click here.…

Continue Reading Vincenzo’s Journey with Glanzmann Thrombasthenia
My Diagnosis Didn’t Silence Me, It Lit a Fire

My Diagnosis Didn’t Silence Me, It Lit a Fire

Editor's Note: Patient Worthy is proud to bring you this story-share, originally written by Gwen Pike, founder of the podcast Empowerment in Illness. When I was in my twenties, life was…

Continue Reading My Diagnosis Didn’t Silence Me, It Lit a Fire
Opinion: Use of Basket Trials to Solve Sleep Problems in Patients with Rare Diseases

Opinion: Use of Basket Trials to Solve Sleep Problems in Patients with Rare Diseases

In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders.  This week…

Continue Reading Opinion: Use of Basket Trials to Solve Sleep Problems in Patients with Rare Diseases
Exercise and Colon Cancer: Can Exercise Reprogram Genes? And Help Colon Cancer Survivors? 

Exercise and Colon Cancer: Can Exercise Reprogram Genes? And Help Colon Cancer Survivors? 

A new study conducted at the Huntsman Cancer Institute in Utah suggests that exercise has a beneficial effect on the body, such as reprogramming, that alters the gene’s function, identity…

Continue Reading Exercise and Colon Cancer: Can Exercise Reprogram Genes? And Help Colon Cancer Survivors? 
Breaking Ground: Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial for QTORIN™ Rapamycin in Cutaneous Venous Malformations

Breaking Ground: Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial for QTORIN™ Rapamycin in Cutaneous Venous Malformations

Palvella Therapeutics, Inc. has announced a key milestone in the development of therapies for rare genetic skin diseases: the completion of enrollment in its Phase 2 TOIVA trial of QTORIN™…

Continue Reading Breaking Ground: Palvella Therapeutics Completes Enrollment in Phase 2 TOIVA Trial for QTORIN™ Rapamycin in Cutaneous Venous Malformations
Diagnosis and management of sleep disorders in Prader-Willi syndrome

Diagnosis and management of sleep disorders in Prader-Willi syndrome

In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders.  This week…

Continue Reading Diagnosis and management of sleep disorders in Prader-Willi syndrome
HYMPAVZI™: A New Era for Hemophilia Patients with Inhibitors

HYMPAVZI™: A New Era for Hemophilia Patients with Inhibitors

Pfizer has announced breakthrough Phase 3 results for HYMPAVZI™ (marstacimab), a once-weekly subcutaneous therapy that could redefine care for people with hemophilia A or B who have developed inhibitors, as…

Continue Reading HYMPAVZI™: A New Era for Hemophilia Patients with Inhibitors
Understanding the Patient Experience with Twice-Nightly Sodium Oxybate Therapy for Narcolepsy: A Social Listening Experiment

Understanding the Patient Experience with Twice-Nightly Sodium Oxybate Therapy for Narcolepsy: A Social Listening Experiment

In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders.  This week…

Continue Reading Understanding the Patient Experience with Twice-Nightly Sodium Oxybate Therapy for Narcolepsy: A Social Listening Experiment
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The Mentor She Wished She Had - How Elizabeth Became a Lifeline for EB
Finding Strength Together: Scott and Katie’s Journey with Advanced Kidney
You Are Not Alone: Empowering the Advanced Kidney Cancer Community
Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
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