A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children's Research Hospital. X-SCID is also known…
According to a story from stuff.co.nz, a baby from Auckland has been diagnosed with two extremely rare disorders that only occur in one in every 10 million people. Her name…
According to a story from Science Alert, the human body is a grand and complex natural structure. While it is easy to think of the body as a single entity,…
9% Copyscape An investigational drug called CLR 131 has received Rare Pediatric Disease Designation from the US FDA for the treatment of Ewing’s sarcoma. The source press release can be…
According to a story from wday.com, Paisley Forsell was what you might call a bit of an adrenaline junkie. She likes to live a high energy, active lifestyle. She loves…
According to information provided by the National Hemophilia Foundation, people living with hemophilia now have the opportunity for a greater level of financial support. This new form of aid comes…
A research team led by scientists from the Beth Israel Deaconess Medical Center (BIDMC) have found that arsenic, in combination with the drug all-trans retinoic acid (ATRA), may have the potential…
According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This…
According to HSC News, researchers at the University of Southern California have engineered a device that may have a sizable impact on those with pediatric hydrocephalus. By troubleshooting the issues…
The award-winning actor Alan Alda has shared his diagnosis of Parkinson’s disease during an interview on the CBS This Morning show. The source video of the interview can be found here on…
A recently published study in the Journal of Biosciences and Medicines has concluded that orthoses may help to improve the gait and balance of patients with Charcot-Marie-Tooth disease. Orthoses are…
A recent study into Periodic Fever, Aphtous stomatitis, Pharyngitis and Adenitis (PFAPA) syndrome investigated the impact of the condition on children and found that is was associated with a reduction…
A recently published paper called ‘Biallelic loss of human CTNNA2, encoding αN-catenin, leads to ARP2/3 complex overactivity and disordered cortical neuronal migration’ outlines new research into the genetic basis of…
According to a story from Gloucestershire Live, Emma Robinson, who is only 46 years old, was just told by her doctors that she has terminal cancer. She had vulvar cancer,…
According to 9News, Harry Campbell was born very premature at 27 weeks. Not only this, but his dimensions were almost unfathomable. Harry was able to fit into the palm of…
Gemphire Therapeutics Inc. has been recommended to stop a clinical trial by the Data and Safety Monitoring Board at Emory University School of Medicine. The clinical trial, which ran into…
Let's take a moment during these dog days of summer to recognize the SMA community during SMA awareness month! Spinal muscular atrophy (SMA) is a rare genetic disorder that causes…
The company Epizyme has announced the results of a Phase 2 clinical study of its lead candidate drug, tazemetostat, in the treatment of relapsed or refractory malignant mesothelioma with loss…
A motor neurone disease clinical drug trial will take place in Scotland for the first time in more than twenty years. This study is the first step in a broader…
The top-line results of a pivotal Phase 3 study investigating the drug recorlevTM (levoketoconazole) in patients with endogenous Cushing’s syndrome have been released. The source article, which contains more detailed information,…
An investigational drug called ALT-P7 is being developed as a potential medicine for certain forms of cancer. The drug has received Orphan Drug Designation for the treatment of gastric cancer…
Orkambi, a medication used to treat cystic fibrosis (CF), has improved the lives of some CF patients since approval last year. CF causes thick mucus that leads to difficulty breathing. …
According to a story from Newswise, an organization called the National Comprehensive Cancer Network (NCCN) has recently released treatment guidelines for a rare type of cancer that can affect women…
According to a story from whtc.com, a recent study from the UK is calling into question the widely held belief that fertility treatments could increase the risk of ovarian cancer,…
According to a story from the University of Toledo, a team of researchers from the school have successfully designed a molecule affectionately dubbed "Sweet-P" that can stop the movement of…
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