Rare Classroom: Cystic Fibrosis
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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How Scent Loss But Recognizing Chili Pepper’s Spice Could Be a Predictor of Parkinson’s
New Recommendations for the Diagnosis and Treatment of MAS, HLH, Interferonopathies
Study Shows One DNA Variant Can Overthrow Harmful Mutations, Explaining Varying Disease Severity
PODCAST: WAIT, HOW DO YOU SPELL THAT?
PATIENT WORTHY SERIES
Study of the Week: Some Genetic Tests Miss Familial Hypercholesterolemia
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: Familial Partial Lipodystrophy
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
Compassion Corner: Should Care be Patient Centric? Does Compassion Matter?
684 words, 9% matched vs 1,329 words, 4% matched Compassion [kuhm–pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by
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.
Focal segmental glomerulosclerosis
Dapagliflozin Benefits Patients with FSGS, Study Shows
From June 5-8, 2021, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Virtual Meeting. Due to COVID-19, the meeting was
Idiopathic intracranial hypertension
IIH Rates are Significantly Higher for Black and Hispanic Women
Some conditions or diseases are more prevalent in certain groups. In the case of idiopathic intracranial hypertension (IIH), new research suggests that it is much
Hydrocephalus
CDI Startup Aims to Address Hydrocephalus
Normally, cerebrospinal fluid (CSF) surrounds our brain and spinal cord, providing numerous benefits from injury protection and nutrient delivery to waste removal. Hydrocephalus, or “water
Phenylketonuria
Study: PKU Patients Face a Higher Health-Economic Burden
Many rare disease patients are familiar with high prices for treatment. In fact, the most expensive medication in the world treats a rare disease: Zolgensa,
Congenital Adrenal Hyperplasia
Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)
Acknowledgment: This story is sponsored by Neurocrine Biosciences and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our
Cystic Fibrosis
Rare Classroom: Cystic Fibrosis
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
Gastroparesis
New Social Media Campaign for Diabetic Gastroparesis Provides Patients Resources and Community
Evoke Pharma focuses on gastrointestinal disease treatments. Eversana helps to provide commercial services for the life science industry. The two companies have just joined together
Ichthyosis
All Patients Enrolled in TMB-001 Trial for Congenital Ichthyosis
In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. (“Timber”) shared that all patients have been enrolled in the Phase 2b
Glioblastoma
AV-GBM-1 Improves Survival in Glioblastoma
Could a specialized cancer vaccine actually improve patient outcomes? According to a news release from biotechnology company AIVITA Biomedical, Inc. (“AIVITA”), the answer could be
Hemophilia
PicnicHealth and Komodo Health Have Combined Their Data to Better Serve Multiple Sclerosis and Hemophilia Patients
Komodo Health and PicnicHealth have just announced that they have partnered together in effort to combine their data and better serve patients. This data will
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