Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
Currently, treatment for multiple system atrophy (MSA) is symptomatic; there are no available therapies designed to slow disease progression. This represents a huge unmet need for this patient community.…
To Diem Nguyen, her daughter Minh is her whole world. Diem would stop at nothing to ensure her daughter gets the help that she needs. That's why, recently, Diem…
In 2020, Anthony Di Laura and Jackie Cucullo were over-the-moon. The married couple had just learned that Jackie was pregnant with their first child. Anthony and Jackie were thrilled…
In a recent news release, clinical-stage biotechnology company Amolyt Pharma ("Amolyt") shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought…
22-year-old Ruchita Sahukari is currently studying Literature and Sociology at the Indira Gandhi National Open University (IGNOU). Her journey to pursuing her degree began with the assistance of non-governmental…
Shortly after he was born, Asa Burnside was diagnosed with Niemann-Pick disease type A, a rare genetic metabolic disorder. Since then, Asa has brought so much joy to his family’s…
In September 2020, Amelia Bradford turned eight months old. Her family loved their fun, wiggly, and happy baby. But then Amelia’s mother Danielle noticed something concerning: a facial bruise around…
Have you ever heard of Orphan Drug designation? In the United States, this designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended…
When Eleni was first born, doctors and nurses told her parents, Neil and Eloise, that their daughter would most likely not survive. That’s because Eleni had been diagnosed with…
Have you ever thought about walking to raise funds and awareness? Well, at the Twin Cities Team Hope Walk, you can do just that! According to Kare11, this event…
12-year-old Rohit, who hails from Uttar Pradesh, India, has a dream to one day become a singer – and he’s ready to pursue that dream no matter what. Throughout…
When Nicole Croxton and Pete Nappi first learned that they were going to have another daughter, the pair were thrilled. But during the course of Nicole’s pregnancy, doctors filled…
The Marburg virus, caused by Marburg marburgvirus (MARV), was first identified in 1967 after hemorrhagic fever outbreaks occurred in laboratories in Serbia and Germany. Since its initial identification, Marburg…
A few years ago, Dr. Thomas Weimbs, a researcher at the Weimbs Lab of UC Santa Barbara, performed a study on mice models of polycystic kidney disease (PKD). During…
Recently, the International Society on Thrombosis and Haemostasis (ISTH) held its 2022 Congress from July 9th to 13th. During the conference, researchers and other stakeholders discussed research, care, and…
Primary progressive aphasia (PPA) is a rare neurological disorder and form of frontotemporal dementia which affects one’s ability to communicate. In many cases, people develop PPA prior to turning…
Earlier this year, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its International Congress. During the Congress, researchers discussed the latest in research and scientific innovation within…
Currently, there are few therapeutic options for people who become infected with the Nipah virus. Rather, treatment is symptomatic. However, that may soon be changing. According to the European…
Michael Klim was traveling three years ago when he felt like something within his body was wrong. The Polish-born swimmer, who has won two gold medals for Australia while participating…
Early diagnosis and treatment is crucial for patients with primary hyperoxaluria type 1 (PH1). Unfortunately, due to a high prevalence of misdiagnosis, as well as poor therapeutic access in…
One thing is sure: 29-year-old Aimee Read was always a fighter. At just two years old, Aimee was diagnosed with leukemia. After two years of chemotherapy and six months…
Currently, ZYNLONTA (loncastuximab tesirine-lpyl) is approved for the treatment of individuals with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) for which two prior therapies had not worked.…
In the United States, during the drug development and approval process, drug developers are able to request Fast Track designation. This process is designed to facilitate and expedite the…
Medical research and clinical trials are extremely important tools in learning more about specific diseases, as well as understanding and evaluating potential treatments. For example, the Phase 2 VIBRANT…
Those living with rare diseases or conditions often face difficulty in finding treatment options - or even spurring researchers to make headway within their disease states. To combat this and…
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