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, no approved therapies exist for ADNP syndrome, a rare genetic neurodevelopmental disorder. But a group of researchers from the UC Davis MIND Institute are working to change that. According…
At 22 years old, Dan Pezzetta is carving out a name for himself in the chronic illness sphere. When he was born, Dan was diagnosed with aortic stenosis. Although intervention…
Earlier this year, 4D Pharma Plc. ("4D Pharma") experienced a "win" when the FDA approved the company's plans to initiate a Phase 1 clinical trial evaluating MRx0005 and MRx0029 for Parkinson's…
25-year-old Grace Strobel is a boundary-breaker. A few years ago, she and her mother began sharing her story at schools or through speeches. You see, Grace has Down syndrome, meaning…
When two previously independent genes join together, and begin transcribing and translating as if they were one, this is known as a fusion gene. Fusion genes have previously been…
Over recent weeks, stories have emerged regarding the frightening situation in Ukraine. People have lost their lives, their homes, and their sources of income. So what happens to those like…
Did you know that there are six muscles attached to each eye which influence the way the eye moves? When these muscles are properly working, the eyes move together and…
If your life goes awry, do you trust your community to stand by you? Luckily, for 6th grader Ethan Chandler, his Carterville, Illinois community jumped in to help when he…
Have you ever heard of PD-L1? The National Cancer Institute explains that PD-L1 is a protein which acts as somewhat of a “brake” for immune response. Sometimes, cancer cells will…
It's safe to say that Gabe Barajas has never been afraid of a challenge. In fact, he embraces challenges - and the excitement and adventure they bring to his life.…
Marine Corps values include abiding by a code of integrity, respecting others, and respecting human dignity. For veteran John Rubino, this code of conduct helps shape the way he lives…
On March 29, 2022, globally diversified chemical company LG Chem announced via press release that its Investigational New Drug (IND) application was approved by the U.S. Food and Drug Administration…
Currently, there are no cures for idiopathic pulmonary fibrosis (IPF), and the two available treatments aim to reduce lung scarring and symptom progression. However, it is clear that new therapeutic…
To be able to pursue your dreams and passions is nothing short of empowering. But for many people within the rare disease community, there are barriers to pursuing our goals.…
In the years leading up to menopause (perimenopause), migraines and headaches become increasingly common. Pair this with mental fogginess and memory issues, changes in mood, altered eyesight, sleep difficulties, and…
In the past, filgotinib (Jyseleca) has largely been used to treat patients with rheumatoid arthritis (RA). However, researchers hypothesized that the treatment could be beneficial in other indications. Following…
In the past, there has never been an approved therapeutic option for those with acid sphingomyelinase deficiency (ASMD). This meant that there was a huge unmet need within this patient…
For the first time, a team of researchers measured heart rate variability (HRV) in those with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) to understand how the autonomic nervous system (ANS) responded…
When Melinda Jansen van Vuuren was 13 years old, she began smoking cigarettes. For years after, Melinda smoked up to 15 cigarettes daily. Although she has since quit smoking, Melinda…
Previous studies have shown that preterm infants (babies born before 37 weeks of pregnancy) often have vitamin D insufficiency. According to Medical Dialogues, researchers in Korea sought to understand the…
Without treatment, an estimated 20% of those with progressive keratoconus may eventually require corneal transplantation. Glaukos, a company creating novel therapies for chronic eye diseases, developed the iLink corneal cross-linking procedure to…
The American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting took place this year from February 25-28, 2022. During the meeting, stakeholders gathered to discuss new technologies, healthcare reform,…
Since its founding in 2009, The Assistance Fund (TAF), an independent charitable patient assistance organization, has helped patients and families within the rare disease and chronic illness sphere with financial…
Unintended weight loss is a symptom associated with many different forms of cancer. But what can this weight loss tell researchers about the patients and their outcomes? Healio shares a…
ProQR Therapeutics is working to develop unique therapeutic solutions for genetic eye diseases. Currently, the company is evaluating its therapeutic candidate ultevursen within the Phase 2/3 Sirius and Celeste clinical trials; you can read…
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