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
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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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Rhabdomyosarcoma- embryonal
Ari’s Bears Provides $30,000 for Rhabdomyosarcoma Research
Childhood cancer is an area that requires research and innovation, as there are currently many unmet medical needs within these patient populations. Fortunately, one non-profit
Alpha-1 Antitrypsin Deficiency
New Treatment For Alpha-1 Antitrypsin Deficiency Lung Disease Has Been Granted Orphan Drug Designation
Mereo BioPharma Group has just announced that they have received Orphan Drug Designation from the FDA for their new treatment for alpha-1 antitrypsin deficiency (A1AD)
Niemann-Pick Disease
Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 1)
“Hi, I’m Rachael, and I’m forever 33.” When Debbie Kaflowitz remembers her daughter, she thinks about everything that Rachael loved to do. Rachael enjoyed dancing
Renal Cell Carcinoma
Study: Cabozantinib for Renal Cell Carcinoma Brain Metastases
A recent study was published in JAMA Oncology outlining a retrospective cohort study analyzing cabozantinib as a treatment for renal cell carcinoma that has metastasized to the
Chronic Inflammatory Demyelinating Polyneuropathy
First Patient Dosed in China Portion of Efgartigimod Trial for CIDP
In mid-November 2021, global biopharmaceutical company Zai Lab Limited (“Zai Lab”) shared via news release that the first patient was dosed in the Greater China
Tuberous Sclerosis Complex
The TSC Navigator: A New Resource to Guide Families through the TSC Journey (Pt. 1)
The TSC Alliance® first began as a grassroots organization, founded by motivated mothers whose children had been diagnosed with tuberous sclerosis complex (TSC), a rare
Multiple system atrophy
Teacher Raises Awareness of Multiple System Atrophy
Nancy Green and Judi Beckerleg first met nearly 22 years ago in 1999. After Judi moved to Arizona, she began working as an aide in
hepatic porphyria
The Porphyria Symposium: Claire’s Experience, and How Conferences Can Better Incorporate The Patient Perspective
Claire Richmond lives with acute hepatic porphyria (AHP). She searched for 19 years to find answers to her unexplained pain. She wasn’t diagnosed until she
Systemic mastocytosis
Study Finds Systemic Mastocytosis Patients Without CMIHR History Don’t Necessitate Premedication Prior to Imaging
Results from a study on systemic mastocytosis (SM) have recently been published. This investigation aimed to understand the prevalence of contrast media induced hypersensitivity reactions
Williams Syndrome
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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