Happy April! We hope everyone's month is going well! This week, we're highlighting two patient stories and two research stories. First, we have the story of Carolyn, a pemphigus patient…
According to a story from Parkinson's News Today, a recent study found that treatment with an experimental gene therapy called AX0-Lenti-PD was able to improve symptoms for two patients with…
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OncLive recently published the views of several speakers at its State of the Science Summit on breast cancer. Stephanie LaBomascus, MD gave a presentation that highlighted triple negative breast…
According to a publication from World Pharma Today, the 16th annual BioAsia conference saw the greatest ever amount of participation at what is consistently Asia’s largest biotechnology and life science…
At the 2019 annual meeting of The Endocrine Society, ENDO 2019, results from a recent study evaluating burosumab for the treatment of X-linked hypophosphatemia (XLH) were presented. This drug was…
According to a story from BioSpace, the genomic medicine company Sangamo Therapeutics, Inc. and Pfizer, Inc. recently announced the release of interim data from a Phase I/II clinical trial. This…
The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the USFDA based on interim results of the clinical trial ADMIRAL according to an article in EurekAlert for Science News. A total of 371 adult patients…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. When: Saturday, April 27, 2019 Where: West Penn Hospital 4800 Friendship Avenue Pittsburgh, PA 15224 Wintergarden Conference Center – First…
Nonalcoholic fatty liver disease (NAFLD) is a condition in which the buildup of fat in the liver poses health concerns. Nonalcoholic Steatohepatitis (NASH) is a more severe, and more rare…
If you haven’t heard of bullous pemphigoid, you’re not alone, the condition isn’t typically well known even by physicians. For those living with it, this tends to mean a long,…
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A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
Cardiff University in Wales has just announced the launch of their new Medicines Discovery Institute. It’s goal? To help accelerate the development of novel therapies for those living with conditions…
The task of letting go starts at the time of diagnosis of the rare disease, when families have to let go of the dreams and expectations they had for the…
According to a publication from FOXBusiness, Waltham, Massachusetts-based Thermo Fisher Scientific Inc. has agreed to buy out Brammer Bio for $1.7 billion. With the acquisition, Thermo Fisher, a lab equipment…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
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A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
According to a publication from Medical News Today, in animal studies, a failed cancer drug showed signs of promise in treating frontotemporal dementia. Frontotemporal Dementia Dementia is a blanket term…
Zachary and Imogen Jones are brother and sister with cystic fibrosis (CF). While Imogen is older, she received her CF diagnosis after her brother. It was actually through educational programs…
According to a story from Parkinson's News Today, April is Parkinson's Disease Awareness Month. This year, special focus will be given to patients with the rare, early onset form of…
According to a publication from EurekAlert, results from a phase 1b clinical study of experimental glioblastoma “vaccine“ IGV-001 suggest that the treatment is well tolerated in humans. Additional data collected…
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According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited. The second finding…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
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Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
According to the publication from RedlionTrader, a second man infected with HIV seems to have been cleared of the infection, those scientists remain highly cautious of touting the case as…
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