According to a story from Angioedema News, the UK's National Institute for Health and Clinical Excellence (NICE) has officially recommended the drug lanadelumab (marketed as Takhzyro) for coverage under the…
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Richard Engel’s son Henry, age 4, has been diagnosed with Rett syndrome, a thus far incurable genetic neurological disease. Engel is chief foreign war correspondent at NBC and has…
A study record from clinicaltrials.gov is investigating the appearance of lung problems in patients with disorders affecting connective tissue. While prior research has documented the appearance of interstitial lung disease…
According to a story from Juvenile Arthritis News, a recent study has found that the drug leflunomide (marketed as Arava) may be a viable treatment for juvenile idiopathic arthritis patients,…
Happy Thursday everyone! This week, we're highlights two stories about patients taking action to solve problems in their life: one patient innovates to improve their care, and another starts a…
According to a story from BioSpace, a recent study of cancer in children is emphasizing the fact that genetic testing is an essential component of cancer treatment, providing critical information…
According to a story from PharmaTimes, the gene therapy company Bluebird bio has released updated results from a clinical study of its Lenti-D gene therapy as a treatment for patients…
According to a story from BioSpace, the biotechnology company BioMarin Pharmaceutical Inc. has recently announced its submission of a clinical trial application with the UK's Medicines and Healthcare Products Regulatory…
According to the British publication The Sun -- a woman's rare disease diagnosis slowly saw her nose collapse - and her nostrils close completely. Following a seizure, Savannah-Kelly Simmonds was diagnosed…
According to a story from Pompe Disease News, a recent study has concluded the polymorphisms of the ACE gene (which are genetic variations that occur naturally) do not have an…
First, I want to be clear-- I'm neither a doctor nor astrologer. This listicle is just for fun! Rare diseases are not Zodiac signs, and are tremendously challenging situations to…
It's common knowledge that your Corona is doing your liver no favours. Heavy alcohol consumption isn’t great for your health. Reported in Cell Metabolism, new research gets more specific: the…
According to a story from Pompe Disease News, a recent study has found that delays in diagnosis are closely connected to whether someone displaying symptoms of Pompe disease is referred…
According to a story from Parkinson's News Today, a phase 2 clinical trial that will test a gene therapy for advanced Parkinson's disease has begun recruiting patients. The experimental gene…
Although even a regular video of twin baby girls snuggling can be engaging and cute, there are two twin girls in the UK whose bond will warn your heart.…
According to a story from sectorpublishingintelligence.co.uk, the drug company Ovid Therapeutics Inc. has recently announced that the early data from its phase 2 clinical trial is mostly encouraging. This clinical…
As originally reported in Biobuzz, the story of rare disease treatment took a turn in the age of modern technology. Rare diseases have traditionally been inaccessible to the naked eye,…
Alnylam Pharmaceuticals has developed and produced a cartoon series aimed at educating people -- especially young people -- about the pediatric rare disease primary hyperoxaluria type 1 (PH1). The campaign…
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A molecular biologist, Dr. Daria Julkowska, coordinates an international consortium in Paris that is largely funded through the 28-member European Union (EU). According to a report published in the Charcot-Marie-Tooth…
According to a publication from Augusta University, a recent study lung cancer patients receiving chemotherapy provided a possible explanation for some cancers' ability to resist treatment. The research, conducted by…
According to a story from Angioedema News, a recent study demonstrated the effectiveness of the medication Haegarda in reducing the number of swelling attacks experienced by hereditary angioedema patients. Preventative…
According to a story from Myeloma Crowd Crowdcare Foundation, the very first Plasma Cell Disorder Congress recently took place in Charlotte, NC from August 10-11, 2019. The event primary focused…
According to a story from ProPublica, the computer servers that store important patient medical data are surprisingly unprotected. This lack of protection appears to be a problem around the world,…
According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics Inc. recently announced that has received a letter from the US Food and Drug Administration (FDA)…
According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…
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