Editor’s Choice: Duchenne, GSD and Orphan Drugs
Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen…
Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen…
Happy Friday! Did you know Patient Worthy has a podcast? In the latest episode, we discuss rare disease dermatology with Dr. Steven Chen, MD of Harvard Medical School. Pemphigus and…
Happy Friday! It may not be a white Christmas for everyone, but with parts of the U.S. receiving upwards of four feet of snow last week, it's close enough. Speaking…
Happy Friday! November 1 is International Acromegaly Day. For those unaware, Acromegaly is a rare condition characterized by the presence of an elevated level of growth hormone and typically effects…
Happy Friday! October is going by quickly, but if you need something else to help pass the time, why not give our podcast a listen? The two newest episodes of,…
Happy Friday! October is Dysautonomia Awareness Month. Dysautonomia is a collection of conditions that affect the autonomic nervous system. This can lead to a loss of control of the body's…
Happy Friday! September is a packed with rare disease awareness, as we've been featuring all month long. We wanted to make sure to draw attention to Dystonia Awareness Month, which…
Happy Friday! Sept. 24 is FH Awareness Day. Familial hypercholesterolemia is a gentic disorder characterized by the inability of the body to remove LDL -- sometimes called "bad cholesterol" --…
Happy Friday! Sept. 13-19 is Balance Awareness Week. It's intended to bring attention to balance-related vestibular disorders such as Meniere’s disease and vestibular migraine. Looking for ways to help out?…
Happy Friday! Monday, Sept. 7 is Duchenne Muscular Dystrophy Awareness Day. DMD is characterized by the inability to make the protein dystrophin in muscles and can result in heart and…
According to a story from The Garstang Courier, Joshua Brandwood of Lancaster, UK has begun organizing a 5K run as a fundraiser for the family of Lexi Tindale, a girl…
Happy Friday! We're about halfway through Stevens-Johnson Syndrome Awareness Month. It's a type of severe skin reaction that often involves admission to a burn unit for treatment. Looking for ways…
Happy Friday! We're about halfway through Stevens-Johnson Syndrome Awareness Month. It's a type of severe skin reaction that often involves admission to a burn unit for treatment. Looking for ways…
Happy Friday! Did you know that August is Stevens-Johnson Syndrome Awareness Month? It's a type of severe skin reaction that often involves admission to a burn unit for treatment. We've…
Happy Friday! July is almost over, which means that Juvenile Idiopathic Arthritis Awareness Month is drawing to a close. But it's not too late to bring attention to this often…
Happy Friday! July is almost over, which means that Juvenile Idiopathic Arthritis Awareness Month is drawing to a close. But it's not too late to bring attention to this often…
Happy Friday! We're more than halfway through July. Where is the summer going? As a reminder, it's Juvenile Idiopathic Arthritis Awareness Month and if you're looking for ways that you…
Happy Friday! The summer heat is setting in, so why not stay in where it's cool and read the latest rare disease news. First, we want to bring attention to…
Happy Thursday! This edition of the Patient Worthy Editor's Choice is coming to you a little early this week. If you're reading from the United States, we hope you enjoy…
Happy Friday! The world could always use more good news, and this week we've got some new discoveries to discuss. Up first, details on a trial that may have finally…
Happy Friday! The world is full of difficult news right now. If you're looking for ways to educate yourself on some of the problems facing the world right now, especially…
Happy Friday! If you're observing Memorial Day, enjoy the upcoming long weekend. Going to use the time to brush up on a little rare disease news? We've got three articles…
One of the goals of treatment for a rare form of non-Hodgkin lymphoma is the delay of disease progression. As reported by Cancer Updates, Research and Education (CARE), a recent…