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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

Eosinophilic Esophagitis

If you have EoE, try Making and Enjoying These Healthy Homemade Foods free from Preservatives and Other Harmful Ingredients

In a previous post, I shared what it took to heal my eosinophilic esophagitis. In this post, I share a few tasty and healthy foods,

Necrotizing enterocolitis

CZI Granted $1M to UNC Researchers to Study Necrotizing Enterocolitis

Prematurity and formula-feeding heighten the risk of necrotizing enterocolitis (NEC), a serious gastrointestinal issue. However, NEC is still poorly understood; in many cases, the

Amyotrophic Lateral Sclerosis

2023 Doddie Aid Event Raises Over £1M for MND/ALS

Doddie Aid, which begins January 1st and continues for the following six weeks, aims to encourage people to get active while raising funds to support

MPS III

Phoenix Nest Launches Natural History Study of Sanfilippo Syndrome

Have you ever heard of a natural history study? In short, a natural history study studies people who have, or are at risk of

Multiple Sclerosis

BRIUMVI Now Approved for Relapsing Multiple Sclerosis

There is no cure for multiple sclerosis (MS). However, treatment advances have helped people to live longer, happier lives. Current therapeutic options include corticosteroids,

Cystic Fibrosis

KB407 for Cystic Fibrosis Granted Orphan Status in Europe

In 2020, the U.S. FDA granted Orphan Drug designation to KB407, an investigational gene therapy for patients with cystic fibrosis (CF). More recently, at

MPS III

Michigan Family Shares Son’s Experience with Sanfilippo Syndrome

Seven years ago, David and Amber Negele married. The pair couldn’t wait to begin their blissful wedded life together. Just a few years after their

Psoriatic Arthritis

Informing Treatment Decisions for Psoriatic Arthritis: A Discussion with Dr. Soumya Chakravarty

From November 10-14, 2022, various stakeholders within the rheumatology sphere connected at the American College of Rheumatology’s (ACR) Convergence 2022. During ACR Convergence, described as

Facioscapulohumeral muscular dystrophy 1a

AOC 1020 Granted Fast Track Designation for FSHD

A January 2023 news release from biopharmaceutical company Avidity Biosciences, Inc. (“Avidity”) shares that the company’s therapy, AOC 1020, earned Fast Track designation from the

Lupus

Balancing CXCL5 Levels Could Potentially Treat Lupus

Currently, the standards-of-care for lupus include anti-malarial drugs such as hydroxychloroquine, corticosteroids and immune-suppressive therapies, and biologics such as Benlysta. But new therapies are urgently