A Phase 2 clinical trial which sought to evaluate the safety, efficacy, and tolerability of NP-120 (ifenprodil) for idiopathic pulmonary fibrosis (IPF) had some surprising results. Initially, the study…
Dr. Cecilia Chung and associates at the Vanderbilt University’s Medical Center conducted a study published recently in MedPage Today. Results showed that patients who had inflammatory disorders, as well…
According to a recent article, the parents of a boy with epilepsy were told they would need to prepare for him to have end-of-life care after they were unable…
Clinical trials are an important tool in medical research to understand more about the safety, efficacy, and tolerability of therapeutics - and how these may influence patient outcomes. Recently,…
Currently, treatment for multiple system atrophy (MSA) is symptomatic; there are no available therapies designed to slow disease progression. This represents a huge unmet need for this patient community.…
To Diem Nguyen, her daughter Minh is her whole world. Diem would stop at nothing to ensure her daughter gets the help that she needs. That's why, recently, Diem…
In 2020, Anthony Di Laura and Jackie Cucullo were over-the-moon. The married couple had just learned that Jackie was pregnant with their first child. Anthony and Jackie were thrilled…
Continued From Part One Written by Becky Tilley Finally I found a role that I was appreciated in, at least by the kids. I wasn't very welcome by the girls…
Scientists have known for years that Wiskott-Aldrich syndrome (WAS) is caused by a gene on the X chromosome. The gene (the WASP gene) can be found throughout the immune…
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…
In a recent news release, clinical-stage biotechnology company Amolyt Pharma ("Amolyt") shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought…
Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…
22-year-old Ruchita Sahukari is currently studying Literature and Sociology at the Indira Gandhi National Open University (IGNOU). Her journey to pursuing her degree began with the assistance of non-governmental…
Written by Becky Tilley My name is Becky Tilley and I am a rare woman, mama, writer and advocate. Also am proud wife to my husband Carl and mother to…
Shortly after he was born, Asa Burnside was diagnosed with Niemann-Pick disease type A, a rare genetic metabolic disorder. Since then, Asa has brought so much joy to his family’s…
In September 2020, Amelia Bradford turned eight months old. Her family loved their fun, wiggly, and happy baby. But then Amelia’s mother Danielle noticed something concerning: a facial bruise around…
Have you ever heard of Orphan Drug designation? In the United States, this designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended…
Danielle Morley of Radcliffe, UK, has been an advocate for plasma donation ever since being diagnosed with a rare blood disorder called immune thrombocytopenic purpura (ITP) during her first…
When Eleni was first born, doctors and nurses told her parents, Neil and Eloise, that their daughter would most likely not survive. That’s because Eleni had been diagnosed with…
Have you ever thought about walking to raise funds and awareness? Well, at the Twin Cities Team Hope Walk, you can do just that! According to Kare11, this event…
As time runs out, volunteer Dominic Gillen is running even harder. In an interview with KETV, he admits that they are not yet where they should be in order…
12-year-old Rohit, who hails from Uttar Pradesh, India, has a dream to one day become a singer – and he’s ready to pursue that dream no matter what. Throughout…
According to a story from Medpage Today, patients using the drug azathioprine who carried a certain genotype were three times more likely to need to either cease treatment or reduce…
When Nicole Croxton and Pete Nappi first learned that they were going to have another daughter, the pair were thrilled. But during the course of Nicole’s pregnancy, doctors filled…
The Marburg virus, caused by Marburg marburgvirus (MARV), was first identified in 1967 after hemorrhagic fever outbreaks occurred in laboratories in Serbia and Germany. Since its initial identification, Marburg…
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