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PATIENT WORTHY SERIES

Study of the Week: A New Treatment Option for Lupus?

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

Rare Classroom: Alternating Hemiplegia of Childhood

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

GACI/ARHR2 Awareness Day is February 1st: Spreading rare Disease Awareness

Generalized arterial calcification of infancy (GACI) and autosomal recessive hypophosphatemia rickets type 2 (ARHR2) Awareness Day is recognized each year on Feb 1. This is

Study of the Week: A new Approach to Treat Mitochondrial Disease

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

fibrodysplasia ossificans progressiva

Rare Disease Patients in Bangladesh Struggle with Diagnosis and Treatment

Mazidul Islam told the Daily Star that he had to watch his 14-year-old son Farid die and could do nothing to save him. Among other

BRCA-mutated Breast Cancer

Antonio Cancer team Finds new Approach for Triple Negative Breast Cancer

An antidepressant drug that was approved by the FDA in 1959 is being put to new and promising use to fight hard-to-treat breast cancers. The

Dr. Rajeev Raghavan is a doctor who works with patients with lupus. He stands in a white lab coat with a light blue shirt.

COVID-19

Dr. Rajeev Raghavan Discusses Data on Lupus and COVID-19 (Pt. 1)

It’s no secret that the COVID-19 pandemic significantly changed the way we interact with the world around us: in our social lives, the way we

Netherton Syndrome

Netherton Syndrome: The Second Clinical Study Has Been FDA Approved to Begin Testing For This Rare Skin Disease

Michael Myers, M.D., CEO of the Quoin pharmaceutical company describes Netherton Syndrome as a devastating skin disorder that at times may be fatal. In a

Progeria

Study of the Week: Scientists Identify new form of Progeria

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

Primary Biliary Cholangitis

Obeticholic Acid Resolves Cognitive Deficits from Primary Biliary Cholangitis

Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently

Beta Thalassemia

Promising Research Results from the 64th ASH Annual Meeting

The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and

Charcot-Marie-Tooth disease

Charcot-Marie-Tooth Patient Overcomes Her Disability by Sailing Solo Around the Globe

Jenny Decker of Fenton, MO. interviewed with KMOV4 explaining that she was unable to walk when she was three years old due to a rare

Fabry Disease

Fabry Disease: Sangamo Therapeutics Announces Updated Data from its Clinical Trial

Sangamo Therapeutics, with its primary focus on genomic medicine, recently issued a news release announcing data from its phase 1/2 STARR trial. The data evaluated

Lymphoma

Zanubrutinib Shows Benefits for Marginal Zone Lymphoma, Study Shows

In 2021, the U.S. Food and Drug Administration (FDA) approved zanubrutinib (Brukinsa) for adult patients with relapsed or refractory (R/R) marginal zone lymphoma. At the