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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

Cutaneous T-cell Lymphomas

Rare Classroom: Cutaneous T-Cell Lymphoma

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

CDKL5

Finding Balance with CDKL5 Deficiency Disorder: Whitney and Havilah’s Story (Pt. 2)

Before you read on, don’t forget to check out Part 1 of the story. In Part 1, Whitney talks about what CDKL5 deficiency disorder (CDD) is and

Rare Disease

FY23 Appropriations Act Includes Increased Funding and Provisions for the Rare Disease Community

On December 23, 2022, President Joe Biden officially signed the Consolidated Appropriations Act, 2023. This bill sets in stone the federal government’s budget for the

Multiple Myeloma

New Immunotherapy Proves Successful in 73 Percent of Multiple Myeloma Patients, (Part 1)

Part 1 Newswise recently published exciting news of a therapy that causes the immune system to destroy cancer cells in the bone marrow. Researchers at

Alex and her son Raymond, who has VAMP2, in 2022. Alex is wearing a white shirt and is kneeling in the grass. Raymond is in a blue shirt and stands in front of her.

VAMP2 disorder

A Ray of Sunshine: Alex Gaudlap’s Efforts to Raise Awareness of the Ultra-Rare VAMP2 (Pt. 1)

In the United States, rare diseases are defined as those affecting fewer than 200,000 people. In the cases of ultra-rare conditions, there is often even

Granulomatosis with Polyangiitis

Study Shows that Rituximab Improves GPA Remission

Determining the safest, best, and most effective therapeutic options for patients is crucial to improving outcomes and ensuring optimal health. As shared in a

Breast Cancer

Why Does Hard-to-Treat Breast Cancer Not Respond to Immunotherapy?

Currently, there are a number of available therapeutic options for those living with breast cancer: radiation, chemotherapy, hormone therapy, targeted therapy, immunotherapy, and surgical interventions.

Microcephaly

Research Explores Two Genes Linked to Microcephaly

As our understanding of genetics continues to grow, new gene variants are being identified and linked to the development of various diseases. According to Medical

Glioblastoma

Could “Cooling” Glioblastoma Improve Overall Survival?

Unfortunately, the prognosis associated with glioblastoma is poor; some studies suggest that survival rates are 40% within one year of diagnosis and 17% within two

Waldenstrom Macroglobulinemia

This May Be the First Published Report on Waldenstrom Macroglobulinemia in Latin America

Waldenstrom macroglobulinemia (WM) is defined as a B-cell lymphoplasmacytic lymphoma. Data from patients in Latin America on WM are scarce. Researchers are hoping to