According to a story from biopharmadive.com, the field of gene therapy has gained increasing relevance throughout 2019, and 2020 will likely be a continuation of this trend. An area of…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
According to a blog post from Visante, concerns from patient groups and advocates about the safety of a recently introduced tube feeding connector device called ENfit have caused major consternation…
Eva Batista is just three months old, and her parents describe her as an "expressive, smiley, and happy baby." Eva is one of the youngest people to ever be diagnosed…
According to a story from tradeshownews.vporoom.com, the Muscular Dystrophy Association (MDA) has recently announced the official approval of its Patient-Focused Drug Development (PFDD) meeting by the US Food and Drug…
An amendment is being planned for an ongoing Phase 1/2 clinical trial for amyotrophic lateral sclerosis (ALS). This intention has just been announced by the cell therapy company Kadimastem. However,…
It wasn’t until the 1980s that the medical field really started talking about the need to emotionally support patients, especially those with chronic illnesses. This is partly due to the…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently granted supplemental approval to the drug acalabrutinib (marketed as Calquence) as a treatment for…
When Lucinka was born, it seemed like everything was all right. But it wasn’t. Lucinka wasn’t doing well, and a whirlwind of examinations began. First, a diagnosis couldn’t be made.…
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Amber’s determination to find a miracle for her son Maxwell is detailed in a recent BuzzFeed article. Maxwell and his twin sister, Riley, were born in March 2017. For…
Narcolepsy is characterized by drowsiness, often during the daytime. This is a symptom that greatly affects the lives of those with narcolepsy, and for a long time no FDA…
According to a story from gurufocus.com, the biopharmaceutical company Scholar Rock has recently announced the release of preliminary results from a phase 2 clinical trial. This clinical trial is testing…
Gastric electrical stimulation (GES) has long been suggested as a treatment for symptoms of gastroparesis, but the use of the technique has been deemed controversial. A recent study published…
When you know your days are numbered, you might stop to think how you want to spend those last days. At times, medical emergencies can put patients into conditions or…
According to a story from Juvenile Arthritis News, the results of a recent phase 3 clinical trial indicate that the drug tofacitinib (marketed as Xeljanz) can be an effective treatment…
The Bettuchi's Amanda Bettuchi and her husband have 3 children. Their middle child, Shane, was born with severe combined immunodeficiency (SCID). Essentially, this means he was born with practically no…
According to a story from Neurology Advisor, the results of a recent study suggest that the presence of psychiatric issues in patients with idiopathic intracranial hypertension is connected to a…
Unfortunately, there are downsides to a lot of treatments for various diseases, such as price, frequency of doses, and discomfort. Any one of these factors can discourage people from…
According to a story from People, Will Yun Lee plays Dr. Alex Park on the medical drama television series The Good Doctor. Although he isn't a doctor in reality, Mr. Lee has…
According to a story from Soweto Urban, mother Lebohang Leuta is no stranger to the challenges that come with raising and taking care of a child with a chronic rare…
According to a story from EurekAlert!, a collaborative study headed by researchers from Yale University has furthered the understanding of the mechanisms and progression of fibrosis (scarring) in patients with…
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An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…
According to a story from The Squamish Chief, mother Emily Tomlinson is amazed by the capabilities of her three year old son Nash Smillie. Nash was born with a severe…
Scientists have been studying animals in order to better understand certain diseases for years. In fact, a genetic disorder that causes blindness called Leber's congenital amaurosis was found in…
For people with sickle cell disease, a common, painful symptom is vaso-occlusive crisis, which is when blood circulation is blocked by sickled red blood cells. This restriction of circulation results…
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