The National Organization for Rare Disorders (NORD) is hosting its 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit on October 21-22 in Washington, D.C. NORD is a great organization and…
Talk about a bittersweet symphony! Published on Yale University's online paper, recent grad Jordan Plotner -- who is living with Ehlers-Danlos syndrome (EDS) -- organized a virtual musical ensemble with other…
The FDA announced last week that it has approved Rituxan (rituximab)to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children 2 years of age and older in combination…
According to a story from healthbeat.spectrumhealth.org, Tasha Veinbergs was 13 years old when she was first diagnosed with Pompe disease. She was visiting the doctor to treat a small cyst…
When you have a rare disease, you know that the way it effects your life goes far beyond the symptoms listed on WebMd. It riddles through plans for weekend, your…
According to a story from American Nurse Today, the US Food and Drug Administration (FDA) approved a treatment on September 6th, 2019 for interstitial lung disease associated with scleroderma, a…
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…
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 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 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 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)…
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