As my children are getting older they seem to create elaborate stories of why they didn’t clean up their room or complete any other task on their to-do list. I’ve…
According to a story from the National Center for Advancing Translational Sciences (NCATS), research supported by the center is making it possible for kids born with serious, rare genetic disorders…
Netflix debuted a documentary series this month called 'Diagnosis' -- based on a New York Times Magazine column -- which follows individuals with or caregiving for patients with a mysterious,…
According to a press release from Eiger Biopharmaceuticals, the American Food and Drug Administration (FDA) recently granted the Company's experimental hepatitis delta virus drug (hepatitis D), peginterferon lambda, breakthrough therapy…
Aevolve, a medical innovation platform, announced last week that it will be making rare disease research a top priority in its blockchain-powered accelerator, which provides a pathway for alternative funding…
It was announced late last week that Justice Ruth Bader Ginsburg had just completed three weeks of radiation treatment for tumors on her pancreas. Pancreatic cancer occurs in the pancreas, an organ…
According to a press release from Synlogic Therapeutics, the Company recently halted development of its investigational hyperammonemia treatment SYNB1020 after an unsuccessful phase 1/2 clinical study. About Hyperammonemia Hyperammonemia is…
Glioblastoma Glioblastoma is a rare type of brain cancer. Sadly, it is the most aggressive form. The median life expectancy for the disease is between 11 and 15 months and…
According to a story from abc.net.au, an unusual and poorly known rare disease has been making its presence known in the country of Australia. This disease is called mal de…
According to a story from Charcot-Marie-Tooth News, a recent study has revealed that patients with Charcot-Marie-Tooth disease (CMT) type 1A still display signs of muscle weakness even if they retain…
Cure GM1 Foundation is hosting a family meeting filled with hope on Friday, September 20th! This gathering will be focused on community, clinical development and research updates, patient advocacy, and…
Families in England affected by Batten disease are struggling to access effective treatments in their home country. Nicole and Jessica Rich are two young girls who live with their families…
TGIF! Something we believe is that the stories of rare disease patients are vitally important. This week, we're highlighting two articles written by patients, as well as a story about…
According to a story from news-medical.net, a recent study is giving scientists new insights into the nature of Merkel cell carcinoma, a rare cancer that affects the skin. The study…
According to a story from Comic Sands, mother Alison Beesley is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
Scientists have discovered an immune suppressing cell to combat symptoms of multiple sclerosis. In a new study on mice, researchers have discovered a cell that can possibly be used to…
According to a story from Angioedema News, the drug development company Regenxbio has announced that it is in the process of developing a new gene therapy treatment for hereditary angioedema,…
According to a story from Scleroderma News, a recent study has recommended that scleroderma patients with a rare, poorly understood form of interstitial lung disease (ILD) should receive regular monitoring…
According to a story from The People's Pharmacy, the health dedicated website (along with many other sites that discuss drug effects and side effects) has experienced a nearly 50 percent…
According to a publication from EurekAlert, a recent study helmed by the Royal Marsden NHS Foundation Trust suggests that female cancer patients tend to live slightly longer following treatment than…
When Amber Freed and her husband Mark resorted to an in-vitro fertilization (IVF) baby they were overjoyed when they found out they were pregnant and were welcoming two new…
According to a story from outsourcing-pharma.com, the company Iqvia has just launched its all-new patient portal. However, while many patient portals are intended to allow for patients to monitor their…
According to a story from The Indian Express, early treatment and diagnosis of primary immunodeficiencies makes management of this group of disease much simpler as a whole. In children, the…
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A study appeared recently in the Orphanet Journal of Rare Diseases reporting that rare diseases affect 350 million people worldwide. The definition of rare disease differs between the United States…
According to a story from kfor.com, Governor Kevin Stitt of Oklahoma recently held a signing ceremony for a bill that is being hailed as a rare example of bipartisan collaboration…
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