Rare Classroom: Stevens-Johnson 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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ALS Drug Facing Possible Rejection by the European Medicines Agency
- June 9, 2023
New Updates are Available on a Phase 2 Trial Evaluating CS1 for PAH
Experimental Therapy for Hemophilia Meets Phase 3 Trial Endpoints
A Newly Initiated Phase 1 Study Will Evaluate AIV007 for Wet AMD, DME
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PATIENT WORTHY SERIES
World Orphan Drug Congress USA 2023: The Forefront of Rare Disease Drug Development
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD.
Rare Community Profiles: How the Seena Magowitz Foundation is Working to Change the World for People Living with Pancreatic Cancer
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease
Study of the Week: RNA Sequencing Platform Could Improve Pediatric Rare Disease Diagnosis
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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Powassan virus
Someone in Maine Died from the Powassan Virus
Ticks can carry a number of disease-causing pathogens. If an infected tick bites a human, that human can then contract several illnesses. In many
Mitochondrial Disease
A High-Efficiency Assay with Rapid Mitochondrial Disease Diagnosis
In Japan researchers at the Juntendo University have discovered that when ECHS1 enzyme variations of mitochondrial enoyl-CoA hydratase short chain 1 (ECHS 1) do
Castleman Disease
Drug Repurposing: Saving Rare Disease Patient Lives
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no
Rare Disease
The FDA Has Approved Ayvakit for the Treatment of Indolent Systemic Mastocytosis
The FDA has approved the drug Ayvakit developed by the biotechnology company Blueprint Medicines for the treatment of indolent systemic mastocytosis. According to an article
Rare Disease
Rare Community Profiles: The Cost of Preventive Care in Chronic Illness and Rare Disease—and Why We Need to Talk About It
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease
Ankylosing Spondylitis
Rare Classroom: Ankylosing Spondylitis
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
Rare Disease
RNA Sequencing: Hope for Improved Rare Disease Diagnosis
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions.
Rare Disease
RDLA Webinar: Mental Health and Rare Diseases
On May 18, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Patient Dies in Gene Therapy Trial
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother
Homozygous Familial Hypercholesterolemia
Study Suggests Lomitapide Could Help Manage Pediatric HoFH
The 91st European Atherosclerosis Society (EAS) Congress took place from May 21-24, 2023. During the Congress, many stakeholders discussed the latest developments in basic, translational,
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