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…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
Patient Worthy recently had the privilege to view a selection of short films about rare disease at Biotech Week Boston. These films highlight the stories of families and patients as…
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…
Kristin woke up the morning of her 19th birthday with a startling realization: she was unable to speak. It was hard to describe the feeling, but when she tried to…
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…
September will soon come to a close, but did you know that this month is also Ovarian Cancer Awareness Month? According to a story from MSN, Michelle Barrenechea, an EMT…
On September 21, 2021, Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. The focus of this webinar was newborn screening in recognition of Newborn Screening Awareness Month, which is…
According to a story from MSN, Busola Omiyale's daughter Toriayooluwa was only nine months old when she could tell that something wasn't right. The girl had sickle cell disease, a…
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…
Jeff was first diagnosed with systemic mastocytosis early in life, at age 16. The first sign that Jeff knew that something was off was when he developed urticaria pigmentosa, abnormal…
According to a story from news.cision.com, the US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for CS1, an investigational drug being developed by the…
According to a press release from Business Wire, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive opinion in regards to the…
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 story from ANCA Vasculitis News, the biopharmaceutical company ChemoCentryx has recently made an amendment to its approval application for its investigational therapy. This therapy, called avacopan, is…
In a recent press release, the US Food and Drug Administration (FDA) has authorized a third dose of the Moderna or Pfizer COVID-19 vaccine for people living with compromised immune…
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…
According to a story from Targeted Oncology, the US Food and Drug Administration has given Orphan Drug designation to ARX788, an experimental therapy. The designation was given for the treatment…
According to a story from News Nation USA, eight-year-old Lilly-Ann Woolliss of Immingham was diagnosed with phenylketonuria (PKU) when she was born. As a result, she must consume a diet…
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) has recently approved a new treatment for late-onset Pompe disease. The drug, developed by the biopharmaceutical…
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…
On September 2, 2021, Patient Worthy attended a webinar from the SYNGAP Research Fund titled "Treatments in Development for Epilepsy Syndromes: Opportunities for SYNGAP1." This program provided an overview of…
When Alison was just nine months old, she was diagnosed with a mild case of cerebral palsy. To this day, she and her doctors still aren’t certain if that's what…
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…
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…
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