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.
In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which…
Personable. Loving. Impactful. Those are all words that Angie Kane would use to describe her son Colton, who passed away at 21 years old in October 2021. When Angie talks…
For the last two and a half years, the COVID-19 pandemic has dominated the headlines. It has been hard to look online – or do anything in person – without…
Have you ever heard of azithromycin? It’s an antibiotic. Chances are, if you’ve ever had a bacterial infection, you’ve even taken it. But according to Medical XPress, researchers are currently…
Jeremy Schreifels was just a young child when he was first diagnosed with Tourette syndrome, a nervous system disorder characterized by sudden, involuntary movements or vocalizations (“tics”). In Jeremy’s case,…
It is very rare that one might say that a car accident saved their life – but Yolanda Carusciello, who lives in Brooklyn, can say just that. Of course, when…
Earlier this year, the U.S. Food and Drug Administration (FDA) approved mitapivat (PYRUKYND) for the treatment of patients with hemolytic anemia due to pyruvate kinase deficiency (PKD). According to 2…
This year, the American Academy of Dermatology (AAD) Annual Meeting took place from March 25-29, 2022. During the meeting, researchers and stakeholders discussed trends and research within the field of…
What type of cognitive changes are present in individuals with spinal muscular atrophy (SMA) type 3? More so, do these cognitive changes affect clinical factors and, if so, how? According…
If you've been paying attention to the news lately, you've probably heard of a rare viral illness that has been spreading around the globe: monkeypox. While monkeypox is more common…
When Brianna Terry was just two years old, her doctors gave her a diagnosis of autosomal recessive osteopetrosis. At the time, the doctors were concerned about her wellness; they…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare conditions. A rare disease or condition is defined as…
In the past, there have been a variety of research studies exploring and working to learn more about cystinuria, a rare genetic condition causing cystine, an amino acid, to accumulate…
Sometimes, it can be difficult to prompt the research of and creation of therapeutics for rare diseases. This is one of the reasons why the FDA created its Orphan Drug…
In 2022, the 25th Annual Meeting of the American Society of Gene & Cell Therapy took place from May 16th-19th. During the meeting, researchers and other stakeholders discussed the latest…
Two years ago, in 2020, life changed for Ian Foster, then 57. At the time, Ian started experiencing health problems and was eventually diagnosed with kidney failure. According to The…
It’s no secret that living with a rare or underserved condition can come alongside a heavy financial burden. A recent study supported by the National Institutes of Health (NIH), Cure…
On May 26, 2022, biopharmaceutical company Nurix Therapeutics, Inc. ("Nurix") shared via news release that positive data was available from an ongoing Phase 1a dose escalation study. Within the study,…
In April 2022, Dr. Adrián Alambiaga successfully defended his doctoral thesis at CEU Cardenal Herrera University. His thesis centered around the exploration of progesterone as a way to stop cell…
In Part 1 of our interview, we discussed Katharina's story, her diagnostic journey to Adult-Onset Still's Disease, and why she was driven to found Perrarus. For Part 2, we will…
Altogether, there are multiple subtypes of spinal muscular atrophy (SMA), a rare genetic disorder causing muscle weakness and generation. Four months ago, Aniya Porter was born with type I, a…
In a news release from late May 2022, pharmaceutical company SteroTherapeutics LLC, known for developing orphan drugs for rare conditions, shared that its novel therapy ST-003 received Orphan Drug designation…
Katharina Clark has always wanted to make a difference. For as long as she can remember, Katharina has held a keen interest in the healthcare sector. Before she was diagnosed…
In the past, medical research has shown that the STAT3 protein plays a role in regulating inflammatory responses throughout the body; it also plays a role in cell proliferation,…
When Hailey Huddleston was just two years old, she had her first seizure. At first, her parents – Tim and Lisa – weren’t sure if Hailey was having a night…
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