According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…
The Rare Diseases Clinical Research Network (RDCRN) has recently launched a survey aiming to assess the impact of COVID-19 on the rare disease patient community. This initiative is important as…
Cures Within Reach is an organization that aims to help patients by repurposing research. They work towards getting treatments quickly and safely in an effort to aid those who need…
Earlier this year, The Leukemia & Lymphoma Society hosted a presentation by Dr. Eunice Wang, Clinical Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center. Dr. Wang’s presentation…
Last week, I had the privilege of attending a webinar organized by Research!America titled Partnership, Progress, Pandemic: The Impact of COVID-19 on Medical Discovery. As implied in the title, the…
June 23rd, 2020 will be recognized as Dravet Syndrome Awareness Day, and June is Dravet Syndrome Awareness Month. These events are dedicated to spreading awareness about the disorder among the…
“The allergist I have here in town thinks I am *overreacting*. You mean, overreacting when my neighbors next door are cooking their meat on the grill and when I walk…
RARE on the Road: a Virtual Rare Disease Leadership Tour June 23rd-24th, 2020 The Virtual Rare Disease Leadership Tour is designed to give insights and education to rare disease advocates,…
According to Myasthenia Gravis News, CAAR T-cell therapy may be a viable treatment option for those with myasthenia gravis. Researchers from the University of Pennsylvania, Yale Medical School, and Cabaletta…
During this time of COVID-19, many people have some general information about COvID-19 including the knowledge that individuals over 65 years of age are at higher risk of contracting…
According to a story from ajmc.com, a recent study found that a new combination treatment for systemic amyloid light-chain (AL) amyloidosis is more effective than the current standard treatment. The…
Parkinson's News Today recently published an article featuring a study in Italy showing the benefits of deep brain stimulation in patients with late-stage Parkinson’s disease. The history of the subthalamic…
According to a recent article in PubMed, immune checkpoint inhibitors (ICIs) are the most commonly used type of cancer immunotherapy. ICIs have brought about a substantial improvement in the prognosis…
Over 2018 and 2019, BioCryst Pharmaceuticals conducted two surveys to better understand the needs and expectations of hereditary angioedema (HAE) patients. Overall, they found that despite progress in HAE therapies,…
In the geographically small island nation of New Zealand, there are estimated to be as many as 300,000 people who live with various rare diseases or disorders. Unfortunately, like in…
According to the ASCO Post, researchers have discovered genomic characteristics of smoldering multiple myeloma that can tell one's risk of progression to multiple myeloma. Dr. Mark Bustoros and his team…
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Summer is approaching, and Lyme disease is coming with it. Ticks and wildlife are more active in the warmer months, leading to an increase in tick-spread diseases. But because we…
According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…
June 17th is recognized as World CDKL5 Awareness Day and the month of June is considered CDKL5 Awareness Month. First organized by the International Foundation for CDKL5 Research (IFCR), the…
A study conducted by researchers from Aix-Marseille University in France has revealed a new way to track disease progression of Charcot-Marie-Tooth disease type 1a (CMT1a)- changes to the nerves within the thigh.…
Celldex has just released positive results from a Phase 1 trial of an investigative therapy which could benefit patients living with chronic urticarias. This disease can result in decreased energy,…
A study led by Yoon Ho Sup, a Professor at NTU Singapore, and Kwang-Soo Kim, a professor at Harvard University, has uncovered two compounds with the ability to increase the…
By Danielle Bradshaw from In The Cloud Copy Angelman syndrome is a rare genetic disorder that is a combination of multiple physical and mental disabilities. 1 in every 15,000 people…
According to Healio, US veterans affected by pulmonary arterial hypertension are typically not treated with exercise rehabilitation, despite the fact that many medical professionals believe that utilizing exercise rehabilitation in…
Five years ago, Leah Gaitan-Diaz's life changed when she was diagnosed with myasthenia gravis. But with this condition comes a lack of understanding or confusion over what it entails. In…
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