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?
PATIENT WORTHY SERIES
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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Acute hepatic porphyria
Canadian Agency Give Positive Opinion for Reimbursement of GIVLAARI for AHP
GIVLAARI has already received approval for the treatment of acute hepatic porphyria (AHP) in Canada, according to an article from Nasdaq. Now, the Canadian Agency
Alpha-1 Antitrypsin Deficiency
Alvelestat for A1AD Earns Orphan Drug Status
According to a late October 2021 news release from biopharmaceutical company Mereo BioPharma Group plc (“Mereo”), the company’s therapeutic candidate alvelestat received Orphan Drug designation
Amyloidosis
New Machine-Learning Model Shows Individuals If They’re at Risk of Rare Cardiomyopathy
According to a recent article, investigators created a machine learning model in order to identify if patients are at risk of rare cardiomyopathy. Transthyretin Amyloid
Carcinoid Syndrome
November is Carcinoid Cancer Awareness Month
A recent article highlighted the fact that November is carcinoid cancer month and is helping spread awareness about the disease in the hopes of earlier
Renal Cell Carcinoma
Study: Tivozanib As a Treatment for Advanced Renal Cell Carcinoma
Recently the Kidney Cancer Association held its International Kidney Cancer Symposium, where medical professionals and other industry members come together to share ideas and push
Oligodendroglioma
Community Rallies Behind Mom with Oligodendroglioma
A Tyler, Tx artist, mother, wife, and friend has her community rallying behind her in her fight against oligodendroglioma, a primary central nervous system cancer.
Cancer
Compassion Corner: Clinical Trial Participants Give Doctors Lower Scores Based on Delivery of Unfavorable Diagnoses
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
Alzheimer's disease
A Drug Shows Amyloid Reduction in Early Alzheimer’s
According to a recent article in Biospace, Eisai Pharmaceutical and Biogen Biotechnology recently announced updates to their Phase IIB study 201 and a five-year open-label
gastric cancer
Will Nivolumab Combined with Chemotherapy be the New Star in Controlling Advanced Gastric Cancer?
Platinum-based chemotherapy has been the standard treatment for patients with metastatic gastric adenocarcinoma for over ten years. According to a recent article in Healio, the
Esophageal Cancer
Toripalimab Snags Orphan Drug Designation for Esophageal Cancer
Orphan Drug designation in the United States is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions, defined as
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