Rare Classroom: Williams 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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“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 2)
- December 1, 2021
Study for Vision Loss from NF1 Optic Pathway Glioma Funded By Gilbert Family Foundation
Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 3)
RGX-202 for DMD Earns Orphan Drug Designation
PODCAST: WAIT, HOW DO YOU SPELL THAT?
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Rare Classroom: Acalvaria
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
Science Simplified: What is Gene Therapy?
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works
Study of the Week: The High Cost Burden of Rare Diseases
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
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Malignant pleural mesothelioma
Durvalumab Hopes to be Treatment Option for Malignant Pleural Mesothelioma
According to a recent article from OncologyLive, investigators are looking at durvalumab to aid in the treatment of adults with malignant pleural mesothelioma in the
Rare Disease
Metastatic NSCLC Survival Boosted by Dual Immunotherapy
According to a recent article, the overall survival of patients with untreated metastatic non-small cell lung cancer (NSCLC) were significantly improved by the addition
Vasculitis
Males With ANCA-Associated Vasculitis Not at Higher Risk of Death or Kidney Failure
According to a recent article, a study showed males diagnosed with ANCA-associated vasculitis (AAV) are not more at risk than females for kidney failure or
Nontuberculous Mycobacteria (NTM) Lung Diseas
First Patient Enrolled in NUZYRA Trial for NTM Lung Disease
On October 18, 2021, biopharmaceutical company Paratek Pharmaceuticals, Inc. (“Paratek”) shared via news release that the first patient had enrolled in a Phase 2b clinical
Rare Disease
Science Simplified: What is Gene Therapy?
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works
Anti-glomerular basement membrane disease
COVID-19 May Be a Trigger for the Development of Anti-Glomerular Basement Membrane Disease
According to a recent journal, a case study found that the development of anti-glomerular basement membrane disease (anti-GBM) may be triggered by COVID-19. Anti-Glomerular Basement
Rare Disease
Study of the Week: The High Cost Burden of Rare Diseases
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
POTELIGEO Beneficial for CTCL, Real-Life Data Shows
In a recent news release from global specialty pharmaceutical company Kyowa Kirin International PLC (“Kyowa Kirin”), the company announced the release of new real-world data
Von Willebrand's Disease
New Study Looks At Bleeding Patterns of Infants and Toddlers With von Willebrand Disease
According to a recent article, researchers reported their findings from a new study that looked at the bleeding patterns and characteristics in infants and toddlers
obstructive sleep apnea
BAY2253651 Ineffective for Obstructive Sleep Apnea
Clinical trials are fantastic ways to develop a deeper understanding of a specific disease, as well as determine the safety, efficacy, and tolerability of potential
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