According to a recent article in MedicalXpress, researchers at Houston Methodist have reported acute transverse myelitis (ATM) in forty-three COVID-19 patients across twenty-one countries. Spinal cord lesions developed in these…
Last April, 25-year-old Obang Odol Okello became infected with dracunculiasis, also known as Guinea worm disease. According to Thomson Reuters Foundation News, the father of two, who lives in Ethiopia,…
A team of great researchers need your help! While the researchers who devised this survey (see below for the link) have had a great response from people with Mal de…
As reported in Gloucestershire Live, Chantelle Parry has a few years head-start on an experience people emerging from this pandemic with symptoms of long COVID may be about to go…
According to a story from MSN, 73 year old Ronnie Wood of the Rolling Stones recently discussed in an interview his struggle with small cell lung cancer during the COVID-19…
Jason Matthews, the author of the award-winning series Red Sparrow and former CIA officer, passed away on April 28th due to corticobasal degeneration. This rare, neurological condition causes the degeneration and…
Recently, researchers sought to examine how Nucala (mepolizumab) affected disease progression in patients with eosinophilic granulomatosis with polyangiitis (EGPA), especially when used in conjunction with corticosteroids. According to ANCA Vasculitis…
According to MedPage Today, ursodeoxycholic acid significantly improves patient outcomes in relation to intrahepatic cholestasis of pregnancy. This rare liver disorder increases the risk of fetal issues, stillbirths, or preterm…
Sanath Ramesh, a software engineer, created the nonprofit Open Treatments after his son was born with an extremely rare condition without any viable treatment options. Raghav, now two and a…
Quarantine during COVID-19 has been difficult for everyone. It's socially isolating, mentally draining, and physically limiting. We all find community different places. But no matter where we find it, it…
Since its inception, the Clinical Data Interchange Standards Consortium (CDISC) has worked to advance data standards to the highest possible quality. These standards are then used to make data more…
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…
Have you been paying attention to the news? If not, you might have missed that INCRELEX (mecasermin) is now commercially available in Canada! In a news release, biopharmaceutical company…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
When it comes to rare diseases, the diagnostic process can be long and arduous. On average, patients wait around 7 years for a diagnosis. Oftentimes, this also complicates treatment and…
One-year-old Alexander was diagnosed with Barth syndrome before he was born; his parents knew to test for the condition after their son Elias passed away from complications in 2018. While…
In a recent news release, biotherapeutics company CSL Behring shared that the company received FDA approval to update the Hizentra label for patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Within…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
Recently, the European Commission (EC) approved a marketing authorization for ADV7103 (Sibnayal), the first approved treatment for patients with distal renal tubular acidosis (dRTA). In a news release, pharmaceutical company…
The month of May is being recognized as Lyme Disease Awareness Month, a time to spread awareness about this disease among the medical field and the general public. As spring…
No one can truly prepare for life raising a child who has a rare disease. But as exhausting as it can be, Lori Dunham describes that raising her little girl…
In this digital age, rare disease issues have become more prevalent and more visible. According to Eurordis, a rare disease affects less than 1 in 2000 people. There are more…
In a news release from April 2021, biopharmaceutical company Jazz Pharmaceuticals, Inc. ("Jazz") shared that the FDA accepted its supplemental New Drug Application (sNDA) for review in relation to Xywav,…
Brickell Biotech has just announced the findings from their Phase 3 study investigating the long-term efficacy and safety of sofpironium bromide gel for the treatment of primary axillary hyperhidrosis. Hyperhidrosis…
The American Academy of Neurology recently held their annual meeting virtually to discuss the latest research in Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS). This meeting was held April 17th through 22nd,…
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