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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A Five Year $2.25M Grant Has Been Awarded for the Study of Alport Syndrome
- December 1, 2021
Study: Telotristat Ethyl Improves Symptoms in Carcinoid Syndrome Patients
Jo’s Story: Receiving a Late Diagnosis of von Willebrand Disease
“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 1)
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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SYNGAP1-related intellectual disability
Boston Children’s Hospital Granted $308,000 Grant by SynGAP Research Fund
According to a recent article from BioSpace, Boston’s Children Hospital was granted $308,000 to advance their multidisciplinary biomarker work. SYNGAP1 Syndrome SYNGAP1 syndrome is a
Alzheimer disease type 3
Scientists Announce Breakthrough Discovery for Alzheimer’s and Other Neurological Diseases
The Times Now News recently featured an article that highlighted a study by a group of NYU Langone scientists. The study paper was published
POTS
How to Protect Your Stomach This Thanksgiving
As someone struggling with chronic illness, you are probably no stranger to the stomach problems that come along with treatment or just a reality of your
Rare Disease
New Partnership Aims to Combat Inequities Faced by Rare Disease Patients of Color
The Rare Disease Diversity Coalition and Global Genes have recently established a new partnership. This partnership aims to help find solutions to help address outcome
Cancer
If Lung Cancer Drug Fails, Takeda Will Offer Rebate
According to a recent article from Medscape, Takeda Pharmaceuticals is offering a rebate for brigatinib (Alunbrig) which is used for the treatment of non-small cell
Multiple Sclerosis
The Similarities and Differences Between Myasthenia Gravis and Multiple Sclerosis
A recent article from Healthline highlighted the similarities and differences between two neurological diseases, myasthenia gravis (MG) and multiple sclerosis (MS). Myasthenia Gravis Deriving from
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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
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