Rare Classroom: 15q24 Microdeletion 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
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New Trial Data Available on Setmelanotide for Bardet-Biedl Syndrome
- September 24, 2021
Chronic Fatigue Syndrome: What Is It and How Does it Affect Patients?
Barth Syndrome Chairman Appeals to the FDA For Appropriate Guidelines When Approving Drugs for Ultra-Rare Diseases
People of Color Are Surgically Treated for HS More Often Than White People
PODCAST: WAIT, HOW DO YOU SPELL THAT?
PATIENT WORTHY SERIES
Study of the Week: A Worm Model of CMTX6?
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
Compassion Corner: This Patient Didn’t Let a Hurtful Incident Ruin Her Appreciation for the Compassionate Care She Received From Other 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 Classroom: Xeroderma Pigmentosum
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
THE LATEST FROM PATIENT WORTHY
paroxysmal nocturnal hemoglobinuria
Study: Pozelimab Shows Positive Results in the Treatment of Paroxysmal Nocturnal Hemoglobinuria
Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria.
Eastern Equine Encephalitis
Eastern Equine Encephalitis Discovered in Mercer County, PA
It’s no secret that mosquitos can carry a variety of viruses and diseases: Zika virus, malaria, Chikungunya, and even the West Nile virus. However, Pennsylvania
Christian syndrome
Brown University Deepens Christianson Syndrome Understanding
Medical research drives a huge amount of our understanding around rare diseases. Recently, researchers from Brown University performed a study to deepen their understanding of
Growth hormone deficiency
New Once a Week Therapy For Growth Hormone Deficiency is FDA Approved
The FDA has recently approved Skytrofa as a treatment for children at least one year of age who are facing growth failure as a result
Sandhoff disease
PLX-200 Earns Orphan Drug Designation for GM2 Gangliosidoses
Within the United States, Orphan Drug designation is granted by the FDA to facilitate the development and review of drugs or biologics intended to
Rare Disease
Pickleball Fundraiser Supports Boy with SLC6A1
When you think of effective fundraisers, do you think of pickleball? Well, a Kansas community did just that. According to a video and transcript on
Crigler Najjar syndrome- type 2
Partnership Develops mRNA-3351 for Crigler-Najjar Syndrome Type 1
From certain partnerships can emerge not only a better understanding of rare diseases but potential therapeutic options. As described by BioProcess International, a nonprofit organization
Sandhoff disease
IB1001 Shows Promise for Tay-Sachs and Sandhoff Disease
Treatment for patients with GM2 Gangliosidosis – which consists of Tay-Sachs disease and Sandhoff disease – has often been symptomatic and supportive. However, a new
Cornelia de Lange Syndrome
This Father is Walking for 1,200 Miles to Raise Awareness for Cornelia de Lange Syndrome
Many rare diseases face issues when it comes to awareness, we know this. Low patient populations, minimal research, and little knowledge all contribute to this
COVID-19
COVID-19 Heightens Preeclampsia Risk, Study Shows
Pregnancy can be stressful enough as is without the looming threat of COVID-19, a novel coronavirus which has played a huge role in our lives
Cholangiocarcinoma
New Diagnostic Tool For Cholangiocarcinoma is FDA Approved
The FDA has recently approved a new diagnostic tool to help determine which IDH1-mutated cholangiocarcinoma patients may be a good fit for the novel IDH1
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