Eliza is now 13 years old and was diagnosed at the age of three with a neurodegenerative disorder known as Sanfilippo syndrome. Eliza’s mother, Dr. Cara O’Neill, a pediatrician and…
Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…
Newborn screening is an important public health initiative that checks for rare and serious genetic, blood, hormonal, or metabolic conditions at birth. The test collects blood through a heel…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
According to a story from the East Bay Times, it was 2003 when Isabel Bueso and her family traveled to the US from Guatemala. She was just eight years old,…
Drug development is a long, expensive, and difficult process. Not every treatment being created makes it to the end of the clinical trial process, much less through FDA approval. Takeda…
When I was a teenager, my mom tried to convince me to become a nurse. As a nurse herself, she told me about the rewards of helping people, the challenges…
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…
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…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
May 15th is recognized each year as Mucopolysaccharidosis (MPS) Awareness Day. The goal of this day is to help spread awareness about this group of diseases among the general public…
By now, everyone has heard of COVID-19. Information is constantly being updated surrounding the nature, symptoms, spread, and potential treatments for the illness. Despite this constant influx of information, there…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
According to a story from gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…
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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
According to a story from globenewswire.com, the biopharmaceutical company Inventiva is scheduled to present the findings from a recent phase IIa clinical trial at the upcoming 16th Annual WorldSymposium™ which is…
A family is grieving for their daughter and taking it one day at a time in the face of a terminal diagnosis. Kelly and Andrew Mills became pregnant with…
Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the…
According to a story from governmenteuropa.eu, Professor Susanne Kircher, who is associated with the Austrian MPS Society and the Medical University of Vienna, writes about the state of rare diseases…
According to a story from Buzzfeed News, Maria Isabel Bueso was born in Guatemala with mucopolysaccharidosis VI, also known as Maroteaux-Lamy syndrome. At age seven she traveled to the US…
According to a story from Benzinga, the biopharmaceutical company Inventiva has recently announced that it has recruited the first patients in a study of biomarkers in Maroteaux-Lamy syndrome. The study…
The New York Times reported that under the Trump Administration's new immigration policy, six migrants undergoing lifesaving treatments have received deportation letters; including a young woman battling a rare disease.…
The Isaac Foundation The Isaac Foundation was established by a man named Andrew McFadyen. He created the organization in 2007 in honor of his son who is diagnosed with the rare…
According to a story from forextv.com, the biopharmaceutical company Seelos Therapeutics, Inc. has recently announced that the US Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND)…
According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China's National Medicinal Products Administration (NMPA) for its…
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