The COVID pandemic affecting eighty-million children in the nation has been a burden on many families. A number that deserves attention is the seven million students who have learning disabilities.…
Zymeworks Inc. has recently began enrollment for an expansion cohort of the Phase 1 trial of ZW49. ZW49 is an antibody-drug conjugate (ADC) that targets HER2 positive cancer cells. This…
In a recent press release, biopharmaceutical company AstraZeneca announced that its drug CALQUENCE (acalabrutinib) met both its primary and secondary endpoints in the Phase 3 ELEVATE-RR clinical trial. These endpoints…
For some parents, learning your child is using cannabis products might cause trouble or upset. Joanne Griffiths is troubled and upset because her son might lose his supply. According to…
Elloise Charles has set out on a mission to raise awareness and money for progressive supranuclear palsy (PSP) after her father was diagnosed. His journey to diagnosis was similar to…
On February 8, 2021, global biopharmaceutical company Bristol Myers Squibb shared that the European Commission (EC) approved a Marketing Authorization Application (MAA) for Inrebic (fedratinib). For around 10 years, there…
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) has approved a new treatment for large B-cell lymphoma that is relapsed or refractory. Refractory means…
Since its inception, biopharmaceutical company Reistone Biopharma Co. Ltd. ("Reistone") has worked to develop treatment options for patients with immuno-inflammatory and autoimmune conditions. Most recently, their drug candidate SHR0302,…
In an early February press release, pharmaceutical company Astellas Pharma Inc. ("Astellas") shared that XOSPATA (gilteritinib) was given conditional approval in China for the treatment of patients with FLT3-mutated…
Note: This press release is from the Association for Creatine Deficiencies. To learn more about this organization, click here. For the first time, a newborn baby has been diagnosed with…
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…
A 31-year-old woman from Kota, Rajasthan has recently undergone a surgery performed partially by a robot, according to The Tribune. The intention of the surgery was to remove two tumors…
On her 23rd birthday, Sara Lefebvre almost died. After having some drinks with friends, her friend drove Sara home. But Sara didn't make it inside; she collapsed in sub-zero temperatures,…
Victoria and Zack Rasberry are asking for a miracle to save their youngest child, Ollie. Ollie and his older sister, Addy, both have metachromatic leukodystrophy (MLD), a rare disorder that…
According to Medical XPress, new research suggests that removing microRNA from triple negative breast cancer cells can reduce the cancer's spread and progression. The research also found that microRNA could…
Improving cognition and identifying abnormal molecular pathways in Alzheimer’s disease has been challenging. Current studies of Alzheimer’s disease have focused on reducing such occurrences as amyloid plaque, neuroinflammation, and neurofibrillary…
February is recognized as Marfan Syndrome Awareness Month, which is an event intended to help spread awareness about Marfan syndrome among the general public and in the medical field. This…
The aHUS Alliance has taken on a new project to raise awareness for atypical hemolytic uremic syndrome (aHUS) and the needs of the patient community, according to aHUS News. The…
The NZ Herald recently published details about the experience of seven-year-old Logan Walsh of Leeds, England. His mother was told to take Logan home after he had been diagnosed with…
Microcephaly is caused by a depletion in neural progenitor cells (NPCs). This depletion is caused by a mutation in the centrosome genes, and it occurs while the brain is still…
In a recent press release, biopharmaceutical company Junshi Biosciences ("Junshi") announced a burgeoning collaboration with leading biologics platform company Coherus BioSciences, Inc. ("Coherus"). The pair is working to develop and…
Last year, 92.7 WOBM reporter Vin Ebenau sat down with Allie and Jonna Crocker, two sisters who live around the Jersey Shore, to discuss their experience with rare diseases. The…
As reported in Charcot Marie Tooth News; researchers have devised a new measure of quality of life for youth with Charcot-Marie-Tooth disease. While of course, researchers could assess the youth’s…
In a recent press release, biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum") shared that the company finished its rolling New Drug Application (NDA) submission. The NDA focuses around maralixibat, an investigational…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
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