Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
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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…
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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…
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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…
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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…
A group of international scientists has been exploring using new DNA-based nanomachines for gene therapy. The nanomachines are hoped to be more effective in the treatment of oncological diseases. As…
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A recent article in the journal Nature reports that Phase 1 GAPVAC-101 First-In-Human trial is one of the first clinical trials with a goal of designing and manufacturing actively personalized vaccines…
M ost of the time, Whitney Weldon doesn’t think about her second skeleton. She was 9 the first time it really flared up. Bridges of bone grew from nowhere to…
418 words 9% vs 744 words 5%Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune DiseasesAccording to a recent article in the EurekAlert researchers used data from…
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A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…
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A recent article in Parkinson’s News Today reports that a reduction in a patient’s required medication and management of motor system disorders may be achievable with Deep-Brain Stimulation (DBS) for…
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Following a record number of approvals in 2018, the FDA has recently approved two new drugs with many more anticipated approvals later in the year. Trazimera to Treat Human Epidermal…
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A recent announcement by the Ehlers-Danlos Society marks the beginning of the recruitment phase for an international study devoted to determining the root cause of hEDS. The gathering of this…
According to a recent article in the Globe, the Danish biotech company Saniona AB has initiated a Phase 2a clinical trial of Tesomet for the treatment of hypothalamic obesity, a rare…
A recent article published in online CANCER NETWORK reports findings that indicate children who have survived Hodgkin lymphoma (HL) may develop solid tumors in later years. These findings will have…
Although some ultrasound and/or CT scan studies may show that patients’ lymph nodes (glands) are free of disease, upon further examination using sentinel lymph node biopsies,(SLNB) the doctors at MD…
Medical devices include ECG electrodes, bandages, cardiac pacemakers, thermometers, and hemodialysis machines. A comparison of clinical trials shows that medical device trials are somewhat similar to pharmaceutical trials that evaluate drugs,…
According to a recent article in the Sunderland Vibe, the MRC Developmental Pathway Funding Scheme (MRC) was created to carry out the work of Professor Roz Anderson, who recently died of…
In the early 1900s, German psychiatrist Alois Alzheimer, while studying about the decline in brain function, recognized a correlation between behavioral changes and the buildup of tangles and plaques in…
Most biological drugs, including insulin, can't be taken orally, because enzymes and proteins cause the drugs to break down too quickly in the digestive system. For decades, the pharma industry…
Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
Massimo Federico, MD, director of the medical oncology unit at the University of Modena and Reggio Emilia, Italy, was quoted in Cancer Network as saying that “Achieving a complete clinical and…
The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…
A recent article in CheckOrphan describes a study that was conducted by Researchers at the Lysosomal and Rare Disorders Research and Treatment Center in Vancouver, B.C. The study examines the…
Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…
Ofatumumab as a Single Agent Results of ofatumumab administered as a single agent with an extended induction (initial treatment) brought about a high response rate and it was well tolerated among follicular…
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