As originally reported in Targeted Oncology, a man was diagnosed with EGFR-Mutated Non-Small Cell Lung Cancer, also known as EGFR-NSCLC. The 60 year old had a previous medical history that…
The world is coming to terms with climate change and the vast number of changes that will come as the earth slowly turns up the heat. A change of this…
As originally reported in the Cincinnati Children's, Brittney writes how she came into the world of rare diseases when her and her husband Michael decided to adopt a baby who…
As reported in Creaky Joints and Healing Hugs Haven; the media has been overtaken by coronavirus on the move. Rare disease patients need to know what that means and what…
1 in 10 people suffer from a rare disease, but when I talk about writing for Patient Worthy, it’s usually the first time people have had a conversation or really…
As originally reported in Biospace, two research networks dedicated to forming bridges between rare disease research around the world will share their niches to promote discovery for rare diseases. TriNetX…
As originally reported in CBC News, the problem wasn't that Tom Salisbury had a cold. The problem was that it wouldn't go away. No rest and patience were enough and antibiotics…
As originally reported in Mercer Island Reporter, Jill Hawkins's three births went smoothly. Her eldest reached expected milestones and began to walk and talk. Her daughter, born four years later,…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
According to a press release from Epizyme, Priority Review has been granted for tazemetostat (Tazverik), a drug used to treat relapsed or refractory follicular lymphoma (FL) for patients whose symptoms…
As originally reported in CNN, a duck billed dinosaur of a time long before humanity has been identified to have suffered from the same rare disease found in children today.…
As originally reported in BioPortfolio, a novel DNA therapy for patients with HPV 6 who experience recurrent respiratory papillomatosis (RRP) has concluded its first clinical trial with promising results. As…
As originally reported in Mayo Clinic, Dr. Veronique Belzil realized she wanted to leave her job as a psychologist to find a cure for amyotrophic lateral sclerosis when she saw…
Having a rare disease means you could have one of any of 5,000-7,000 distinct disorders, each with their own quirks. Just how many there are though remains unclear. As originally…
As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine), has received fast track designation, meaning patients may be able to receive…
As originally reported in StatNews, Mark Zuckerberg, founder of Facebook, along with his wife, Dr. Priscilla Chan, a former pediatrician and now head of the Chan Zuckerberg Initiative, made an…
As originally reported in ArcaMax, when you get a diagnosis for a terminal rare disease, a wave of emotions crashes over life. Brian Wallach emerged clutching 'hope.' In 2017, Brian,…
As originally reported in BioSpectrum; the World Health Organization (WHO) has just approved the disease code for CDKL5 Deficiency Disorder (CDD), adding it to the list used worldwide for the…
As originally reported in Cystic Fibrosis Life Foundation, Martha has spent her 24 years of life with cystic fibrosis. She was born with the rare disease which means for a…
As originally reported in Global Data, the FDA has just given the final approval for a treatment options for pulmonary arterial hypertension (PAH) called 'Bosentan.' Pulmonary Arterial Hypertension Pulmonary Arterial Hypertension…
As originally reported in NewsRoom, the US's political volatility over the past years has thrown many policies and legislation into debate. While switching the party in both the executive and…
As originally reported in PR Week; pharma company Takeda has been joined a plethora of rare patients groups to create the 'I am Number 17 ' campaign which features 17…
As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…
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A medication that works for some patients with a rare disease may not be effective for others, even with the same disease. For rare diseases, this is important because sample…
For much of human history, many genetic disorders were accepted as incurable, their rapid onset bound to take patients' lives before they had the chance to be lived. However, the…
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