According to an article in the Washington Post, a recent comment by a WHO official caused a furious debate and criticism of the organization. Maria Van Kerkhove, the head of…
by Danielle Bradshaw, In the Cloud Copy A common occurrence among children that have rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation or ROHHAD syndrome is that they’re also…
By Lauren Thayer from In The Cloud Copy Latent autoimmune diabetes in adults or LADA is a slow progressing form of autoimmune diabetes. Similar to type 1 diabetes, also an…
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…
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.…
Every day, the world becomes increasingly technological. With this comes advances in many realms, from healthcare to education. Recently, says Parkinson's News, medical technology company Manus Neurodynamica received £750,000 (approx. $950,000)…
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 Cheri Tannenbaum spends her days making 1960s flower child themed clothing and it’s a style that she’s rather fond of wearing too.…
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…
When the 19-year-old French woman first visited the hospital, she was extremely lethargic. She displayed neurological symptoms. Upon kidney failure, the patient was admitted into the ICU. But soon, doctors…
By Danielle Bradshaw from In The Cloud Copy Expressive language sampling, or ELS, could potentially help researchers better measure how well treatments work for patients with fragile X syndrome or…
Arabel Standingbear sketches figures with lead pencils and paints realistic scenes with acrylics. Her poem was accepted by a museum. She has won in local art contests between schools, and…
Nicox SA, an international ophthalmology company, recently announced a Phase 3 clinical trial for NCX 470 to lower intraocular eye pressure. The article recently appeared in Eye Wire News. The…
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…
An estimated 25-30 million Americans are part of the rare disease community. If you're one of them, you share a common (yet uncommon) experience: public scrutiny, difficulties in diagnoses, treatment…
June is recognized as Myasthenia Gravis Awareness Month. Primarily organized by the Myasthenia Gravis Foundation of America (MGFA), this event is focused on spreading awareness as well as boosting efforts…
According to a story from PR Newswire, CureDuchenne Ventures has recently announced the initiation of its CureDuchenne Ventures 2020 Pitch Contest. This marks the first time that the contest has…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its…
According to a story from BioSpace, the biopharmaceutical company MacroGenics, Inc. has just announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's…
Acadia Pharmaceuticals recently released the results of their Phase 3 trial of Nuplazid, a treatment for the psychosis that Parkinson's disease patients experience. This medication has been approved since 2016,…
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