PODCAST: WAIT, HOW DO YOU SPELL THAT?

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

paroxysmal nocturnal hemoglobinuria

ICYMI: Empaveli for PNH Now Approved in Canada

Currently, C5 inhibitors such as eculizumab and ravulizumab are considered the standard-of-care for people living with paroxysmal nocturnal hemoglobinuria (PNH). Unfortunately, not all patients

Microcephaly

Study of the Week: Study Finds Two Genes Related to Microcephaly

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

Perthes disease

Boy with Perthes Disease Forms Bond with Dallas Cowboys’ Cooper Rush

Throughout his life, Gavin Miller has overcome many struggles. As a young child, Gavin always loved hockey. But when he was in a hockey

Alveolar soft part sarcoma

ICYMI: FDA Approves Tecentriq for Alveolar Soft Part Sarcoma

While alveolar soft part sarcoma (ASPS) is considered to be relatively slow-growing, it is necessary to carefully manage this rare soft tissue sarcoma to

Von Willebrand's Disease

ICYMI: VGA039, a First-in-Class Antibody Therapy for VWD, Launched at ASH Annual Meeting

The 65th American Society of Hematology (ASH) Annual Meeting took place from December 10-13, 2022. During the meeting, stakeholders in the hematology field came

Alzheimer's disease

INTERVIEWS: NFL Players Discuss the Rare, Chronic, and Underserved Causes that Mean the Most to Them (Pt. 1)

From September to January each year, our home transforms; each Sunday, my boyfriend and I meticulously set up our living room, make snacks, and spend

food allergies

Seattle Seahawks DE Shelby Harris Shares His Son’s Experience with FPIES

Shelby Harris is no stranger to facing challenges; throughout his football career, Harris has remained as a strong and steady defensive lineman with a career

Multiple Myeloma

New Immunotherapy Proves Successful in 73 Percent of Multiple Myeloma Patients (Part 2)

Continued From Part One Multiple myeloma is caused by the buildup of abnormal white blood cells that form tumors found primarily in bones. Available

hypoparathyroidism

Hypoparathyroidism: Positive Results from Phase 2a Trial

Globe Newswire recently published an article announcing positive results from Amolyt Pharma’s Phase IIa proof of concept clinical trial of AZP-3601. Amolyt Pharma, Cambridge, Massachusetts,

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.

Rare Disease

A Ray of Sunshine: Alex Gaudlap’s Efforts to Raise Awareness of the Ultra-Rare VAMP2 (Pt. 2)

Before you read on, don’t forget to check out Part 1 of our interview. In Part 1, Alex discusses the multi-year diagnostic odyssey to discover that Raymond