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

Charcot-Marie-Tooth disease

“I Was Misdiagnosed with Every Disease That You Can Think of:” A Charcot-Marie-Tooth Disease Story

Written by Lily Sander By the time I was age four, I started experiencing unexplained symptoms such as severely turned in feet, which caused extreme

Pancreatic Cancer

Rucosopasem Manganese Granted Orphan Drug Designation for Pancreatic Cancer

On May 18, 2023, Market Watch reported that rucosopasem manganese received Orphan Drug designation. Developed by clinical stage biopharmaceutical company Galera Therapeutics (“Galera”), rucosopasem

Phenylketonuria

Primary Endpoint Met in Sepiapterin Trial for PKU

Within the Phase 3 APHENITY study, researchers worked to determine the efficacy of sepiapterin in both adults and children with phenylketonuria (PKU). Formerly known

Sickle cell anemia

Hydroxyurea is Recommended for Children with Sickle Cell Anemia. So Why Does It Remain Underused?

In 2014, the National Heart, Lung, and Blood Institute released updated clinical guidelines in reference to the care of children with sickle cell anemia. The

Granulomatosis with Polyangiitis

Damage from Cocaine Abuse can be Misdiagnosed as Granulomatosis with Polyangiitis

The damage caused by cocaine can sometimes be misdiagnosed as a rare disease: granulomatosis with polyangiitis. Cocaine use has been increasing in America and

Neurofibromatosis

Rare Classroom: Neurofibromatosis Type 2

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

Bardet-Biedl Syndrome

Study Shows How Setmelanotide Reduces Hyperphagia Associated with Bardet-Biedl Syndrome

Hyperphagia, or excessive and unrelenting hunger, is a key symptom associated with Bardet-Bield syndrome. This excessive hunger often begins around or before 5 years

Rare Disease

Compassion Corner: Compassion Should be Reciprocal: The Rising Suicide Rate of Physicians and Nurses

Compassion [kuhm–pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the

Rare Disease

myTomorrows Launches Clinical Trial Tool TrialSearch AI

On April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an

Pancreatic Cancer

PYX-201 Granted Orphan Drug Designation for the Treatment of Pancreatic Cancer

Pyxis Oncology is driven to improve the lives of people living with cancer through the development of innovative immunotherapy and antibody drug conjugate (ADJ) therapies.