Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
Last week, biotechnology company Triplet Therapeutics announced their participation in END-DM1, a natural history study to learn more about myotonic dystrophy. There are two major forms of myotonic dystrophy, a rare…
by Danielle Bradshaw from In The Cloud Copy It’s possible for fat to end up stored inside of the liver due to either obesity, diabetes, certain medications, or high cholesterol.…
by Lauren Taylor from In The Cloud Copy Rheumatoid arthritis (RA) is a chronic inflammatory disorder that affects a patient’s joints, in addition to causing damage to the skin, eyes,…
Of the things we take for granted about our existence, I venture to say vision is pretty high on the list. Even if your eyesight starts to go it can…
by Lauren Taylor from In The Cloud Copy Duchenne muscular dystrophy (DMD) is a rare genetic condition that ultimately leads to the wasting away of muscles over time. The condition…
COVID-19 turned our worlds upside down back in March, and it has been the main focus for many medical professionals since. Because it is a novel coronavirus, we did not,…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
28-year-old Melissa Loewen has always enjoyed helping others. It was this spirit of community that led her to a nursing role in the BC Women’s Hospital's NICU. But a few…
According to a recent article in FierceBio, Andrea Mazzocchi, Known Medicine CEO, is not trying to reinvent the wheel. On the contrary, her company will be providing a tool to…
Over the last 2 years, the UK has seen 4 deaths and 320 safety incidents related to missing steroid usage, a story from The Independent explains. For patients with Addison's disease,…
As reported in Crwe World; the biopharmaceutical company Chiasma aims to create oral consumed therapies in order to provide acromegaly patients who have a chronic need for injections with an…
Last week, biopharmaceutical company Deciphera Pharmaceuticals announced results from its Phase 1 clinical trial on QINLOCK (ripretinib). The study, published in the Journal of Clinical Oncology, tested QINLOCK as a potential…
It goes without saying that change is not easy for most people. If life has been a certain way for many years and something alters the course of things, it…
According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…
In our 21st century world, we have become enveloped by technology. In the age of COVID-19, we are even more reliant. In an instant, so many of our every day…
Gary S. Gilkeson Career Development Award (CDA) The CDA is a grant sponsored by the Lupus Foundation of America that was established to help accelerate research for lupus. The 2020…
Living with a rare disease can be difficult. Carlos, who has myositis, knows all about this. His mobility has been declining, and he is now in a wheelchair. His doctors…
In 2012, a new medical technology emerged: CRISPR-Cas9, which offered the opportunity for gene editing. LabBiotech explains that gene editing could benefit patients with genetic disorders by adding, deleting, or…
Recently, biopharmaceutical company BerGenBio ASA ("BerGenBio") announced that their drug candidate bemcentinib reached its primary efficacy endpoint in a Phase 2 clinical trial. The drug is designed to treat patients…
In a recent press release, biopharmaceutical company Aligos Therapeutics announced that the first patient was dosed in their Phase 1a/b clinical trial. The trial is designed to assess ALG-010133 as…
Myeloproliferative neoplasm (MPN) International Awareness Day is on September 10th. This year, there will be a live streaming event to help bring awareness to this condition, instead of an in-person…
According to MedCity News, a third patient dosed with AT132 during the ASPIRO trial has died. The Phase 1/2 clinical trial tested the efficacy, safety, and tolerability of AT132 for…
Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…
BioMarin Pharmaceutical Inc. has recently submitted a new drug application (NDA) for their achondroplasia treatment, vosoritide. This once a day injection is for children who live with the disorder, and…
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