Cushing's disease, like all other rare diseases, benefits greatly from increased awareness and advocacy. Because there are fewer patients and affected individuals, their voices need to be louder in order…
Recent research has given us a better understanding of acromegaly, a rare hormonal disorder. Conducted by Maria Fleseriu, MD, FACE, and colleagues at the Pituitary Center at Oregon Health &…
Did you know that there are six muscles attached to each eye which influence the way the eye moves? When these muscles are properly working, the eyes move together and…
If your life goes awry, do you trust your community to stand by you? Luckily, for 6th grader Ethan Chandler, his Carterville, Illinois community jumped in to help when he…
Since 2015, April 6 has been recognized as Bohring-Opitz Syndrome Awareness Day, a time to spread awareness about this rare disease among the medical field and the general public. The…
Have you ever heard of PD-L1? The National Cancer Institute explains that PD-L1 is a protein which acts as somewhat of a “brake” for immune response. Sometimes, cancer cells will…
CANbridge Pharmaceuticals recently announced via press release that the Taiwan Food and Drug Administration (TFDA) has accepted their New Drug Application (NDA)/Orphan Drug Registration (ODR) for CAN108. Marketed as LIVMARLI,…
It's safe to say that Gabe Barajas has never been afraid of a challenge. In fact, he embraces challenges - and the excitement and adventure they bring to his life.…
Definitive Healthcare launched a survey in November 2021 to help physicians, researchers, and the rare community understand what the biggest challenges are for providing a rare disease diagnosis and treating…
Marine Corps values include abiding by a code of integrity, respecting others, and respecting human dignity. For veteran John Rubino, this code of conduct helps shape the way he lives…
On March 29, 2022, globally diversified chemical company LG Chem announced via press release that its Investigational New Drug (IND) application was approved by the U.S. Food and Drug Administration…
Myrtelle Inc. has just announced that they have been granted three new designations from the FDA for their investigative gene therapy called rAAV-Olig001-ASPA. This therapy is being investigated as a…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Juvena Therapeutics has just announced that they've received a new grant which will fund their research of myotonic dystrophy type 1 (DM1). This was a very competitive grant which totals…
Awareness and advocacy are extremely important to every rare disease, and porphyria is no different. That's why we celebrate Porphyria Awareness Week (PAW) every year from April 2nd until the 9th.…
Currently, there are no cures for idiopathic pulmonary fibrosis (IPF), and the two available treatments aim to reduce lung scarring and symptom progression. However, it is clear that new therapeutic…
According to a recent article, Canadian actor Cameron Mathison opened up about dealing with Legg-Calve-Perthes disease throughout his childhood. Perthes Disease Perthes disease is a condition that affects the hips…
According to a recent press release from Passage Bio, their investigational gene therapy for Krabbe disease, PBKR03, may be ready to move into the clinical trial stage of drug development.…
To be able to pursue your dreams and passions is nothing short of empowering. But for many people within the rare disease community, there are barriers to pursuing our goals.…
In the years leading up to menopause (perimenopause), migraines and headaches become increasingly common. Pair this with mental fogginess and memory issues, changes in mood, altered eyesight, sleep difficulties, and…
In 1873, a Russian physician coined the term “multiple myeloma” after discovering a patient with eight different forms of bone marrow tumors. In the early years, patients were treated with…
Bristol Myers Squibb (BMS) has been developing luspatercept-aamt - marketed as Reblozyl - as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In order to create treatments and diagnostic methods for rare diseases, it's necessary to have a thorough understanding of said disease. A recent study aimed to do just this. Titled…
On March 29, 2022, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar titled "An Overview of Amyloidosis and WM with Dr. Morie Gertz." The goal of this program was…
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