As originally reported in Michigan Medicine News, the Nephrotic Syndrome Study Network (NEPTUNE) has received their third five year cycle of funding to research rare kidney diseases. Right now, NEPTUNE…
As originally reported on NPR, Alex Yiu’s mom has a montage of her son's early years. As one watches the videos, you see something: him losing control. He begins to…
According to a story from BioSpace, Oncotelic, Inc., recently announced the publication of a study in the Journal of Clinical Research in Pediatrics. This study appears to back up the therapeutic…
As a second year pre-medical student at Northeastern University, my desire to become a healthcare professional flourishes every day. An advantage of being a student who is just beginning to…
Niemann-Pick disease type C (NPC) is a rare disease caused by a defected NPC protein. In a healthy individual, this protein helps the body process cholesterol. Those diagnosed with NPC experience…
According to a story from MDMag, the US Food and Drug Administration (FDA) has recently approved a label expansion for the drug treprostinil (marketed as Orentiram). This update to the…
When Jordan Berkhoudkt was in middle school, he began to have pain that they couldn’t find a way to relieve. He couldn’t get an accurate diagnosis from the doctors in…
According to a story from the Borehamwood & Elstree Times, the family of 11 year old girl Sivan Hermon is plagued by a rare disease: primary pulmonary hypertension. As the…
Happy Thursday! This week we're highlighting four patient stories: a woman managing her HAE symptoms, a singer with a scary diagnosis, a Bechet patient building support networks, and a CIDP…
According to a story from Pulmonary Hypertension News, pulmonary arterial hypertension and its variants can ultimately result in a cascade of impacts on other areas of the body, including the…
You may forgive the Sci-Fi esque headline, but sometimes the truth is stranger than fiction! The 28-year-old from France (identified in this CNN piece as Thibault) was almost completely paralyzed…
Do you want to participate in your wellbeing and in your medical care? Do you want your health care providers to share your data with you? Do you want…
According to a story from ir.stockpr.com, the biotechnology company Cyclo Therapeutics, Inc. (previously known as CTD Holdings) has recently thrown its support behind a Niemann-Pick Disease Type C newborn screening…
Hereditary Angiodema Hereditary angioedema (HAE) is a rare disease typically caused by a SERPING1 gene mutation. This protein is responsible for coding for the protein C1-INH. With improper levels of…
According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…
According to a story from Targeted Oncology, recent data suggests that the experimental therapy IMG-7289 could be a useful treatment for patients with myelofibrosis, a rare type of cancer that…
Bohuslav Skoupý had trouble with his kidneys. He even had a transplant. He also had heart problems. In the end, it turned out actually by chance that Fabry disease was…
According to a story from Rare Disease Report, there are several different rare diseases and conditions in which patients can find relief from most or all of their symptoms by…
As originally reported in WWVA, West Virginian Sib Weatherford spent one afternoon this fall doing what nobody though possible: he slid 400 feet down the New River Gorge's high-line in…
About HAE Hereditary angioedema (HAE) is a rare disease that is caused by the deficiency or reduction of a protein called C1-INH in the body. When this protein is not present,…
As originally reported in SF Gate, Jeannette and Victor Vega quickly realized that taking care of their second daughter, Tiana, was not going to be so easy this time. From…
According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…
According to a story from Venture Beat, the company Healx has succesfully raised $56 million in funds that will go towards using artificial intelligence (AI) to identify new potential treatments…
According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…
Gypsy Rose Blanchard spent a life suffering through neverending doctors appointments, surgeries, living in a wheelchair, and being fed through a feeding tub. As it turned out, these treatments weren't…
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