The Burdens of Chronic Disease There are many types of chronic diseases, affecting patients in all kinds of ways. However chronic conditions share one thing in common- they all pose…
Idiopathic pulmonary fibrosis (IPF) is a rare condition with no known cause and no effective treatments besides a lung transplant. Current therapies focus on slowing progression of the disease but…
A new medication called selumetinib has just received Breakthrough Therapy designation by the FDA for pediatric patients diagnosed with neurofibromatosis type 1 (NF1) or inoperable pleciform neurofibromas. This therapy has…
"When only 100 people in the world share your child’s specific gene mutation, it is hard to find anyone who understands, including doctors." Rare diseases are at a disadvantage when…
According to a story from Stockwatch, the gene therapy company bluebird bio, inc. recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…
The FDA has just announced that they've approved Mayzent for the treatment of relapsing multiple sclerosis (MS). Multiple Sclerosis Multiple sclerosis is a neurological autoimmune disease. It causes a wide…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc. recently announced that the US Food and Drug Administration (FDA) has approved the company's application to…
According to a story from Market Screener, the biopharmaceutical company Imbrium Therapeutics LP recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation for the…
According to a story from Euroinvestor, the drug development company Resverlogix Corp. recently announced that it has received the necessary $2.9 million in funding for a Phase 2 clinical trial…
According to a story from businesswire.com, the biopharmaceutical company PellePharm, Inc. recently announced that it has dosed the first patient in the company's Phase 3 clinical trial. This trial is…
Recently Dr. Janet Maynard, who currently is the director of the US Food and Drug Administration's (FDA) Office of Orphan Drugs Development, wrote an article in which she discusses the…
Happy April! We hope everyone's month is going well! This week, we're highlighting two patient stories and two research stories. First, we have the story of Carolyn, a pemphigus patient…
At the 2019 annual meeting of The Endocrine Society, ENDO 2019, results from a recent study evaluating burosumab for the treatment of X-linked hypophosphatemia (XLH) were presented. This drug was…
Nonalcoholic fatty liver disease (NAFLD) is a condition in which the buildup of fat in the liver poses health concerns. Nonalcoholic Steatohepatitis (NASH) is a more severe, and more rare…
Cardiff University in Wales has just announced the launch of their new Medicines Discovery Institute. It’s goal? To help accelerate the development of novel therapies for those living with conditions…
According to a publication from FOXBusiness, Waltham, Massachusetts-based Thermo Fisher Scientific Inc. has agreed to buy out Brammer Bio for $1.7 billion. With the acquisition, Thermo Fisher, a lab equipment…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
Zachary and Imogen Jones are brother and sister with cystic fibrosis (CF). While Imogen is older, she received her CF diagnosis after her brother. It was actually through educational programs…
According to a story from Parkinson's News Today, April is Parkinson's Disease Awareness Month. This year, special focus will be given to patients with the rare, early onset form of…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
A New Partnership Patient Worthy is honored and excited to announce our new partnership with The B.L.A.I.R. Connection. The B.L.A.I.R. Connection was founded by Grey Chapin who knows firsthand what…
If you are an avid follower of the latest news, then you have probably heard about one the latest sensational headlines to come out of DC lately: the US Department…
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