Order Up! How Dr. Consulta is Changing the Culture of Medicine
A Brazilian company known as Dr. Consulta is taking healthcare to a place only previously known by fast food chains. The company, founded in 2011, breaks the mold as a…
Mika: Honest, Whole, and Valuable
One of my better friends within the cystinosis community is Mika Covington. We met at a cystinosis event in 2016. I had read some of Mika’s entries posted in her…
Zero Cater Shares Gut-Friendly Snacks to Add to Your Pantry
We’ve all become a little more in tune to what’s good for our bodies, and what isn’t. Part of that is medical science finally being able to identify our bodily…
Positive Results from Phase 3 Trial for Multiple Myeloma!
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
High-Resolution Genetic Map Shows De Novo Mutations Aren’t Random
deCODE Genetics, which is a research subsidiary of Amgen genomics based in Iceland, has recently created an extremely detailed genetic map. They wanted to uncover more about the human genetic…
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Is The FDA Doing Enough to Regulate the Quality of Generics?
According to a story from The People's Pharmacy, there are an increasing number of worrying stories and incidents that are calling into question the US Food and Drug Administration (FDA)'s…
Editor’s Choice: CIDP Diagnosis Struggles and Rare Cancer Survivors
Hope everyone's week is going well! Today, we're highlighting an article discussing the difficulties of securing a CIDP diagnosis, followed by an update on a Niemann-Pick Type C treatment. Next,…
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Wife Successfully Diagnoses Her Husband’s POEMS Syndrome Before Doctors Did
According to a story from the New York Times, a 50 year old Houston man had seen his health decline rapidly in a little over a year. After declining from…
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Company Plans Extension Study for Phase 3 Trial of Amyotrophic Lateral Sclerosis Drug
According to a story from pm360online.com, the Orion Corporation, a pharmaceutical company based in Finland, has recently announced that it plans to continue its Phase 3 trial of levosimendan as…
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Unusual Bardet-Biedl Syndrome Case Raises Questions About Cancer Risk
According to a story from Genetic Obesity News, the unusual case of a female Bardet-Biedl syndrome patient who also had endometrial cancer is causing researchers to wonder if the two…
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Company Earns Orphan Drug Designation for Autoimmune Hepatitis From the FDA
According to a story from finanznachrichten.de, the Swedish drug company Calliditas Therapeutics has recently announced that the US Food and Drug Administration (FDA) has given one of the company's experimental drugs…
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New Combination Treatment Gains Approval for Chronic Lymphocytic Leukemia
According to a story from Rare Disease Report, the US Food and Drug Administration (FDA) has announced its approval of a combination therapy from Janssen which consists of ibrutinib plus…
Grant will Fund Technology That Could Diagnose Rare Diseases Faster
Mendelian Mendelian is a healthtech company in London whose primary goal is to help the National Health System (NHS) diagnose rare diseases faster. With 3 million people currently living with a…
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Potential Gene Therapy for Late Infantile Batten Disease Earns Rare Pediatric Disease Designation
According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…
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Is it Possible to “Train” the Immune System to Accept Transplanted Organs?
According to a story from independent.co.uk, organ transplants have been responsible for saving many lives. Organ transplants are often a critical part of treatment for a lot of rare diseases.…
Did You Catch Microsoft’s Super Bowl Commercial?
The Super Bowl is obviously a big deal for football fans - but it's also (for a lack of a better term) the SUPER BOWL of commercials. Over 100 million…
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Ulcerative Colitis: Delaying Treatment Could Cost You
According to a story from Medical News Today, leaving ulcerative colitis (a form of inflammatory bowel disease) untreated could result in dire consequences for a patient. While ideally all patients…
Study Shows Treatment may be Effective for Chronic Kidney Disease but Increase Risk of Hyperkalemia
A recent study, called TOPCAT, evaluated the effects of spironolactone as a treatment for individuals living with Chronic Kidney Disease (CKD) who are also diagnosed with heart failure with preserved ejection fraction,…
Importance of Patient-Centered Research in Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome (FCS) Is a rare disease caused by malfunctioning lipoprotein lipase. This results in a buildup of triglycerides in the body's plasma. FCS can cause pancreatitis, memory loss, nerve…
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Hyperkalemia and Medication: Which Drugs Can Cause it?
According to a story from GoodRx, hyperkalemia, which is a condition of elevated levels of potassium in the blood, can be a potentially life threatening situation. The condition can appear…
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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial
According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…
23andMe Just Got FDA Approval for Rare DNA Cancer Test
We've all seen the commercials - especially during the holiday season - for home DNA kits like 23andMe and Ancestry DNA, where a saliva sample can unlock your family heritage.…
Potential New Oral Treatment for Hereditary Angioedema Scores Well in Safety and Efficacy
Hereditary angioedema (HAE) is a rare condition which causes severe swelling in various areas of the body. Takhzyro has been approved as a treatment for this disease. The drug was created…
<a href="https://pixabay.com/users/TheDigitalArtist/">TheDigitalArtist</a> / Pixabay
Fundraiser Held for Boy Diagnosed With Niemann-Pick Disease Type C
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
Bring Awareness to Neuroblastoma on February 2nd
February 2nd is Neuroblastoma Awareness Day! It's important to know more about this rare cancer, since knowledge and familiarity are often the engines of progress. What is Neuroblastoma? Neuroblastoma is a…
