The University of Oxford and University Hospitals in Cleveland, Ohio, today announced a multi-year affiliation to establish a global center for rare diseases. The Oxford-Harrington Rare Disease Centre will bring together the capabilities, resources and expertise of both institutions to deliver new treatments for rare diseases, for which therapeutic options are lacking. More than 350 […]Continue Reading ...
Surrounded by patients who told horror stories of being stuck with hefty bills, President Donald Trump recently waded into a widespread health care problem for which almost everyone — even those with insurance — is at risk: surprise medical billing. Trump’s declaration that taming unexpected bills would be a top priority for his administration echoed […]Continue Reading ...
Researchers have traced the origins of a rare genetic disease to two Quebec founding families in the 17th century There are only 25 people in the whole of Quebec at the moment who have a rare recessive genetic disease called chronic atrial and intestinal dysrhythmia (CAID). It is a serious disease that affects both heart […]Continue Reading ...
New research has shown that many Australians with rare cancers can benefit from genomic profiling. The findings of the patient-driven trial are being presented today at the Clinical Oncology Society of Australia Annual Scientific Meeting and could result in dramatic changes to the way those with rare cancers are diagnosed and treated. The initial data […]Continue Reading ...
Moderna, Inc., a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced new pre-clinical research published in the journal Nature Medicine that further demonstrates the utility of its mRNA platform to express therapeutic levels of protein in liver tissue to potentially […]Continue Reading ...
August 20, 2018 RDMD, a healthcare technology company dedicated to accelerating research for patients with rare diseases, today announced that it has raised $3 million in seed financing. The financing was led by Lux Capital, with participation from Village Global, First Round’s Healthcare Co-Op, Garuda, Shasta Ventures and various angel investors from the biotech and […]Continue Reading ...
Credit: CC0 Public Domain Scientists conducting one of the largest full DNA analyses of a rare disease have identified a gene mutation associated with a perplexing brain condition that blinds and paralyzes patients. A UT Southwestern study that used genetic data from more than 1,200 participants may help scientists improve treatments of neuromyelitis optica (NMO). […]Continue Reading ...
Angiotensin converting enzyme-inhibitors are commonly prescribed to patients with high blood pressure. In rare cases, this type of medication can cause severe, even life threatening, swelling to the head and neck area. Credit: Shutterstock Approximately half a million Danes are currently prescribed some type of ACE inhibitor to reduce their blood pressure. Worldwide, that number […]Continue Reading ...
Even after all she had been through — the helicopters circling her house, the snipers on the roof and the car ride to jail — Lisa Abramson still wanted to have a second child. That’s because right after her daughter was born in 2014 — before all that trouble began — everything felt amazing. Abramson […]Continue Reading ...
Rare cancers inherently make it difficult to collect data, build clinical expertise and treat affected patients – challenges which are best addressed through collaboration in international networks. The European Society for Medical Oncology (ESMO) is therefore launching Rare Cancers Asia (RCA), a multi-stakeholder partnership that will build on the success of the existing Rare Cancers […]Continue Reading ...
Patients with rare diseases often go through a long history of suffering before finally receiving an accurate diagnosis. One reason for this is that the patient information necessary for research exists in varying languages and formats. This is set to change, thanks to a new European Joint Program in which Goethe University plays an important […]Continue Reading ...
AUSTIN, Texas, Oct. 01, 2018 (GLOBE NEWSWIRE) — Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, today announced the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to the Company’s lead product candidate, pegzilarginase, for […]Continue Reading ...
TUESDAY, July 31, 2018 — Azedra (iobenguane) has been approved by the U.S. Food and Drug Administration to treat people 12 and older with rare adrenal gland tumors that can’t be surgically removed and have spread beyond the original site. The adrenal glands sit above the kidneys and produce the stress hormones epinephrine and norepinephrine. […]Continue Reading ...
May 17, 2018 Value in Health, the official journal of ISPOR (the professional society for health economics and outcomes research), announced today the publication of a series of articles offering important insight regarding the challenges in rare disease diagnosis, drug approval, and patient access. The special themed section appears in the May 2018 issue of […]Continue Reading ...
March 20, 2018 In this video, meet participant Alexander Masterson and his mum, Kirsty, who speak about what getting a diagnosis from the 100,000 Genomes Project means to them. Find out about the rare disease that he was diagnosed with as a child, Noonan syndrome, and how his recent genetic diagnosis of LEOPARD syndrome is […]Continue Reading ...
- FDA Approves Zulresso (brexanolone) for the Treatment of Post-Partum Depression
- How it manipulates us to tribalism
- How can doctors encourage patients to adopt healthier behaviors?
- Meet Hal: He's One Sick Robot
- Blood test and mathematical model can estimate preterm birth rate in low-resource countries
- TAVR procedure safe in patients with unusual valve anatomy
- Proteins in the eye may be potential source for cost-effective test to predict Alzheimer’s disease
- Opioid Prescriptions Dropped for New Users From 2012 to 2017
- New method may better predict the best treatment for burn wounds
- “Asian” isn’t specific enough for health data, research suggests
- ColumbiaDoctors Presents Honors for Outstanding Commitment to Patient Safety
- Innovative model identifies primate species with potential to transmit Zika in the Americas
- One-off surgery could offer hope to patients with high blood pressure
- How to Protect Your Kids From Drowning
- CEA Test: MedlinePlus Lab Test Information
- Two years after face transplant, Andy Sandness’ smile shows his progress
- Registration now open for Stanford’s Big Data in Precision Health conference
- Gene Keeps Fear at Bay, But Only in Females
- GARDP and Evotec partner to tackle growing threat of antimicrobial resistance
- Ultrasound offers precise, minimally invasive way to measure cardiac output in children