ALS Drug Facing Possible Rejection by the European Medicines Agency

Rose Duesterwald - June 9, 2023

pulmonary arterial hypertension

New Updates are Available on a Phase 2 Trial Evaluating CS1 for PAH

HemophiliaHemophilia AHemophilia B

Experimental Therapy for Hemophilia Meets Phase 3 Trial Endpoints

Diabetic macular edemaWet macular degeneration

A Newly Initiated Phase 1 Study Will Evaluate AIV007 for Wet AMD, DME

PODCAST: WAIT, HOW DO YOU SPELL THAT?

PATIENT WORTHY SERIES

Rare Classroom: Stevens-Johnson Syndrome

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

World Orphan Drug Congress USA 2023: The Forefront of Rare Disease Drug Development

The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD.

Rare Community Profiles: How the Seena Magowitz Foundation is Working to Change the World for People Living with Pancreatic Cancer

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease

Study of the Week: RNA Sequencing Platform Could Improve Pediatric Rare Disease Diagnosis

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we

Williams Syndrome

Woman with Williams Syndrome Throws First Pitch at Brewers Game

The energy in the crowd at American Family Field was electric. Everybody sat on the edge of their seat, waiting for the game to begin.

Rare Disease

Rare Community Profiles: Uplifting Athletes Harnesses the Power of Sport to Benefit the Rare Disease Community

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease

Spinal Muscular Atrophy

This Spinal Muscular Atrophy Patient Established a Nonprofit for Disabled Artists

John knew from an early age that he not only loved music but had confidence in his musical ability. However, there was another very troubling

Non-Hodgkin lymphoma

First Patient Dosed in ACE1831 Study for NHL

In a late May 2023 news release from clinical-stage biotechnology company Acepodia, the company shared that the first patient has been dosed in a

Rare Disease

Science Simplified: All About Genetic Sequencing

Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works

Dystrophic epidermolysis bullosa

Vyjuvek Now Approved for Dystrophic Epidermolysis Bullosa

Managing dystrophic epidermolysis bullosa (DEB) can be difficult. DEB is a rare genetic condition which causes fragile, easily blistered skin. There are multiple DEB

Diabetic macular edema

Vabysmo Shows Advantages Over Aflibercept in Wet AMD and Diabetic Macular Edema

An announcement from the drug company Genentech highlights the results of a recent study which demonstrated that the drug faricimab-svoa (marketed at Vabysmo) was able

Fabry Disease

Rare Community Profiles: From the Virtual Fun Run & Walk to Mental Health Support: How Jack Johnson of FSIG is Working to Improve the Lives of All People with Fabry Disease

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease