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.
What prognostic tools can be used to assess patients with idiopathic pulmonary fibrosis (IPF)? How can researchers use these tools to determine the efficacy of therapies during clinical trials, or…
When Nikolas and Ryan Michaud think of their little sister Jillian, they think of the joy she brings to their life. Nikolas says that Jillian's hugs are unmatched, maybe the…
In a news release from February 16, 2022, clinical-stage biopharmaceutical company AN2 Therapeutics, Inc. ("AN2") shared that its therapy epetraborole was granted Orphan Drug designation for the treatment of non-tuberculous…
When Sam Anderson was thirteen years old, he was diagnosed with familial adenomatous polyposis (FAP), a rare inherited disorder. As he grappled with his diagnosis and symptoms, Sam also…
On February 16, 2022, Healio shared that CT103A, a therapy for adult patients with relapsed or refractory (R/R) multiple myeloma (MM), had received Orphan Drug designation from the United States…
CFTR gene mutations cause cystic fibrosis, a rare genetic condition which causes progressive digestive and respiratory system damage. Yet it can be extremely difficult to treat for those with “stop”…
An estimated 1 in every 200,000 Americans has lymphangioleiomyomatosis (LAM), a rare lung disease that disproportionately affects females. To this day, researchers are still learning new information about this condition.…
In a news release from early February 2022, clinical-stage pharmaceutical development company Algernon Pharmaceuticals Inc. ("Algernon") shared that its Phase 2 clinical trial evaluating Ifenprodil (NP-120) was fully enrolled. Within…
On February 7, 2022, Healthline reported that the FDA had approved Vabysmo (faricimab-svoa), an injectable therapy for those with wet age-relate macular degeneration (wAMD) and diabetic macular edema. This approval…
If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…
The Ebola virus is endemic in certain areas of Africa, where viral outbreaks still exist to this day. For example, there were at least three Ebola outbreaks in Africa during…
February 15, 2013 marked the first-ever International Angelman Day. The day, still celebrated nine years later, aims to raise global awareness for Angelman syndrome. Additionally, International Angelman Day serves to…
HTT gene mutations cause Huntington’s disease, a rare and inherited neurological condition. Those with a family history of Huntington’s disease may choose to undergo genetic testing to learn more, though…
Have you ever heard of comorbidities? In short, comorbidities mean the presence of more than one disease or condition in someone at a time. For example, arthritis and heart disease…
Clinical trials, drug development, and medical research have the potential to change the therapeutic landscape for patients with a wide variety of conditions. At ProQR, founder and CEO Daniel de…
Digital health company AEYE Health was founded on the desire to provide fully automated, artificial intelligence (AI) based diagnostic screening tools for retinal imaging. According to the Ophthalmology Times, the…
In a news release from late January 2022, biopharmaceutical company CASI Pharmaceuticals, Inc. ("CASI") shared that the Company's partner, BioInvent International AB ("BioInvent") recently earned an Orphan Drug designation from…
Renal cell carcinoma (RCC) is a highly metastatic (easily spreading) cancer. If RCC progresses, it can spread to other organs, such as the lungs. Although RCC is a rare cancer,…
In October 2021, the FDA approved Susvimo (ranibizumab injection) for the treatment of wet age-related macular degeneration. Now, shares WFMZ, Dr. Chin Yee from Georgia Retina has performed the first…
As many of you know, Rare Disease Day 2022 is coming up at the end of the month. Celebrated on February 28, Rare Disease Day aims to raise awareness and…
Orphan drug designation is granted to drugs or biologics intending to treat, diagnose, or prevent rare diseases or conditions. Within the United States, a rare condition is one affecting under…
Newborn screening is a public health program which screens infants for a variety of genetic, endocrine, and metabolic disorders following birth. For example, a newborn screening panel may test for…
The Central Mississippi Down Syndrome Society (CMDSS) is a parent-driven nonprofit organization which serves to provide support for Central Missourians with Down syndrome and their families. In addition to offering…
In a February 8, 2022 news release, late-stage clinical company Synthetic Biologics, Inc. shared that the FDA granted Orphan Drug designation to the therapeutic candidate VCN-01. VCN-01 is a therapeutic…
People with epidermolysis bullosa (EB) often experience symptoms such as intense itching or severe pain. Unfortunately, these symptoms can be debilitating and may greatly affect overall quality-of-life. However, some burgeoning…
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