For decades researchers have had to isolate segments of genes in order to study them. They called the isolated gene a “mini gene.” Ravindra Singh, a biomedical science professor at…
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…
According to a press release from the US Food and Drug Administration (FDA), the agency has approved the medication resmetirom (marketed as Rezdiffra) as the first-ever treatment intended to treat…
Medication can be an essential component of managing attention deficit hyperactivity disorder (ADHD). These medications, such as Vyvanse and Adderall, can help to: Reduce symptoms like impulsivity, hyperactivity, and inattention…
In some cases, healthcare providers can identify Angelman syndrome in utero through a prenatal ultrasound or prenatal genetic testing. But could this open the door to early treatment and better…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
A new global study (THAOS NCT00628745) of over 6,000 people who had been diagnosed with ATTR amyloidosis found that almost one in every four patients exhibited cardiac and neurological symptoms.…
Ultomiris (ravulizumab) and Soliris (eculizumab), both developed by global biopharmaceutical leader AstraZeneca, are both effective treatments for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Yet this efficacy…
When you’re a baseball fan, any chance you get to join in and really experience the game is amazing. Seven-year-old Ella McKee has always been fascinated by baseball. She loves…
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 think is of particular interest…
Vision loss can be challenging. If you could have a one-time gene therapy that would improve your visual acuity, would you take it? It seems like a no-brainer. That's what…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Drs. Julie Gerberding…
Over 100,000 individuals living in the U.S. are on a waiting list for an organ transplant. An average of 17 people die each day while waiting for a donor organ.…
During the past three years two drugs (Aduhelm and Leqembi) that were designed to slow the progression of Alzheimer’s disease, have been approved. A verdict by the FDA on a…
Researchers from the Max Planck Institute for Evolutionary Anthropology (MPI-EVA) have been collecting and analyzing data on ancient DNA for years. This DNA has given the researchers a stronger…
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 some of the rarest, most…
According to a recent article in MedicalXpress, scientists have discovered a possible method of treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study was also published in Science…
Recently, the US Food and Drug Administration (FDA) approved a label expansion for the monoclonal antibody dupilumab (marketed as Dupixent) in the treatment of a rare disease called eosinophilic esophagitis…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Anna-Lena Neehus of the Imagine Institute at Paris Cité University was searching through genomic data from over 15,000 patients. She wanted to understand how and whether genetic deficiencies contributed to…
The European Society of Cataract & Refractive Surgeons (ESCRS) held its ESCRS Winter Meeting from February 15-18, 2024. During the meeting, Dr. M.S. Swathi, MD, shared data from a pilot…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
According to reporting in Science Daily, a team of researchers affiliated with MIT and the University of Helsinki have identified a protein found in human sweat that could offer protection…
The month of April is recognized as Sjögren's Awareness Month, a time for spreading awareness about the autoimmune disease Sjögren's syndrome in the medical community and among the general public.…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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