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…
Sometimes, epidemics don’t make headlines. That seems strange, a major health risk causing death to ripple through communities, and yet we're more likely to hear about whats happening with the…
According to a story from the TGen Blog, a simple typing mistake lead to a diagnosis for a boy with an incredibly rare genetic disorder. In fact, this disorder is…
Happy Thursday! It's cold and flu season around our parts, so we hope everyone reading this has dodged the fall viruses! This week, we're sharing a recap of a Global…
According to a story from news-medical.net, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20 different research teams. The goal?…
According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…
As originally reported in The Age, Karen Carey, 57, and Connie Severino, 42, were called to the emergency department at St Vincent’s Hospital at the same time. Each were told,…
EPP Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come…
According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…
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