November 10, 2022 - Patient Worthy

Photo courtesy of CureDuchenne

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)

Post author:Jessica Lynn
Post published:November 10, 2022
Post category:Duchenne Muscular Dystrophy Timely

Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…

Photo courtesy of Sangamo

Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA

Post author:Jessica Lynn
Post published:November 10, 2022
Post category:Fabry Disease

The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research…

source: shutterstock

OTX-2002 for HCC Earns Orphan Drug Designation

Post author:Jessica Lynn
Post published:November 10, 2022
Post category:Hepatocellular Carcinoma

The Orphan Drug Act was passed in 1983 to stimulate the development of therapeutics for rare diseases. With this came the Orphan Drug designation. This designation is granted to drugs…

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First European Patient Dosed in Berubicin Trial for GBM

Post author:Jessica Lynn
Post published:November 10, 2022
Post category:Glioblastoma

Glioblastoma multiforme (GBM) is notoriously difficult-to-treat. This cancer is both aggressive and invasive, spreading “tentacles” and becoming hard to remove even with surgery. As a result, finding more effective therapeutic…

mcmurryjulie / Pixabay

AVR-RD-02 Earns Rare Pediatric Disease Designation for Gaucher Disease

Post author:Jessica Lynn
Post published:November 10, 2022
Post category:Gaucher Disease

The FDA created the Rare Pediatric Disease Designation and Priority Review Voucher Program to stimulate the development of therapies intended to treat, diagnose, or prevent rare pediatric diseases. These are…

CureDuchenne Has Launched a Certified Occupational Therapist Program: An Interview with Jennifer Wallace Valdes, PT (Pt. 2)

Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA

OTX-2002 for HCC Earns Orphan Drug Designation

First European Patient Dosed in Berubicin Trial for GBM

AVR-RD-02 Earns Rare Pediatric Disease Designation for Gaucher Disease

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

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

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