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

Non-Hodgkin lymphoma

White Sox Pitcher Liam Hendriks Returns to Field After Conquering Non-Hodgkin’s Lymphoma (NHL)

In December 2022, Liam Hendriks—a pitcher for the Chicago White Sox—was diagnosed with non-Hodgkin’s lymphoma (NHL); he shared this news with the world in January

Duchenne Muscular Dystrophy

MP1032 Earns Orphan Drug Designation for DMD

As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage

Right eye retina can help scan for multiple sclerosis

Wet macular degeneration

How Carmen’s Wet AMD Experience Challenged Her to Raise Awareness

At first, the round black dots that Carmen saw spattered across the bathroom sink were frustrating, but not concerning. Since her grandchildren were staying with

MPS VI (Maroteaux-Lamy syndrome)

Rare Community Profiles: “The Boy in the Blue”: An MPS and Mental Health Awareness Project

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

Chronic myelomonocytic leukemia

Early Data Suggests Lenzilumab Benefits for CMML

The AACR Annual Meeting 2023 took place from April 14-19. During the meeting, stakeholders from across the cancer community—from patients to clinicians—come together to discuss

Ovarian Cancer

Ovarian Cancer: Second Patient Treated with Novel CAR-T in Trial

The biotechnology company Anixa Biosciences, Inc. together with the Moffitt Cancer Center, issued a press release on May 22nd stating that the second patient

Acute disseminated encephalomyelitis

Acute Disseminated Encephalomyelitis Can be Confused for a Stroke

Thirty-year-old Brendan O’Reilly was told that his rare disease affects only one person in 250,000. He was also told that he is lucky to

IgA Nephropathy

There Is No Limit to What Some Mothers Will Do For Their Children.

The Chow family was told that their 33-year-old daughter, Brittney, would have to wait about twelve years for a kidney transplant. There were currently no

Chronic Kidney Disease

MRI Can Help with Earlier CKD Identification and Diagnosis

What do clinical evaluations, blood tests, urine tests, kidney tissue tests, and imaging tests all have in common? These are all diagnostic measures that doctors