Six years ago, Jonathan Kowalske's son Mac entered the world - and the family suddenly found themselves thrust into the rare disease community. Soon after birth, Kowalske and his…
Understanding rare, chronic, or uncommon conditions can be difficult. This is especially true when there is a general lack of education and awareness. Dr. Aditi Kantipuly decided to make…
Most people view Christmas as a time for celebrating. Spending time with family and getting out to festive events and parties can be cornerstones of the holiday. For children like…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Over their lifetime, people with neurofibromatosis type 1 (NF1), a rare genetic disorder, have a 30-50% chance of developing plexiform neurofibromas. These tumors grow on peripheral nerve sheaths throughout…
Health professionals continually warn the public to take steps to guard against sunburn. We often hear the expression “one in a million.” The phrase and the warning truly pertain…
The risk of developing an inflammatory bowel disease (IBD) such as Crohn's disease or ulcerative colitis (UC) has been slowly but steadily increasing over time, with an estimated 0.8%…
Unfortunately, Jody McPherson, 47, a veteran firefighter with the Mesquite Fire Department, passed away after his battle with Creutzfeldt-Jakob disease (CJD). As reported by Selena Hernandez of CBS News Texas,…
Roche has added another failure this year with its drug gantenerumab. As noted in Fierce Biotech, the drug was unable to improve functional or cognitive decline in two Phase…
Two emergency surgeries that began 48 hours after birth, meningitis, and over a month in the intensive care units of three Seattle hospitals prompted Layla Babayev’s desperate parents to enroll…
The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food…
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…
Prior to the FDA’s approval this month, nirogacestat (Ogsiveo) received the following FDA designations applicable to the treatment of desmoid tumors: Breakthrough therapy providing a rapid review. Fast track to…
Surgical resection remains the most common treatment approach for people living with tenosynovial giant cell tumors (TGCT). Unfortunately, surgery is not always an appropriate treatment option for all people.…
Written by Martha Harlam My journey with Ataxia has been a life full of ups and downs. Ataxia has defined most of my life since it was first diagnosed in…
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.…
Bacterial meningitis is a serious bacterial infection affecting the meninges (the membranes around the spinal cord and brain). The membranes then swell and press on the spinal cord and…
Meenakshi Rao, an assistant pediatric professor at the Children’s Hospital in Boston, recently expressed his opinion to the RNA-Seq blog that inflammation, not bacteria, should be targeted, as infections…
Written by Maria Palombini Answer: at the time of diagnosis, it should be one of the first actions you, the patient and/or caregiver, should take. Why? Receiving a diagnosis with…
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 a story from BioPharma Dive, the pharmaceutical company Celldex Therapeutics recently released positive results from two clinical trials. These trials involved the company's investigational therapy, barzolvolimab. In a…
Clinical studies are critical tools in identifying and developing therapeutics for a wide variety of conditions. This research is especially important in the rare disease and chronic illness spaces,…
445 words 10% matched vs. 2061 words 2% matched We invented medicines to kill bacteria; now, the bacteria are killing us. Lucien Swetschinski, a researcher and co-author at IHME, told…
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.…
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…
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