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.
According to the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
What do phenylketonuria (PKU), galactosemia, and maple syrup urine disease (MSUD) have in common? All three are considered inborn errors of metabolism, or rare genetic disorders in which the…
Currently, there are few - if any - ways to treat patients with myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS). Rather than treating the condition itself, doctors often treat symptoms…
Have you ever heard of isoflavone? Well, isoflavone is part of a class of phytoestrogens, or plant-based compounds which resemble estrogen. According to Medical XPress, research from Dr. Ashutosh Mangalam,…
Breakthrough Therapy designation is granted by the FDA as a way to accelerate drug development and review. In particular, the designation is granted to drugs or biologics for serious conditions,…
On July 14, 2021, biopharmaceutical company Alkeus Pharmaceuticals, Inc. ("Alkeus") shared via press release that its therapy ALK-001 (C20-D3-vitamin A) received Breakthrough Therapy designation from the FDA for the treatment…
Normally, B lymphocytes ("B cells") are a type of white blood cell that plays a role in immune function. In healthy individuals, B cells create antibodies to fight infections and…
In a news release from mid-July, 2021, biopharmaceutical company Strongbridge Biopharma plc ("Strongbridge") shared the publication of new post hoc analyses regarding KEVEYIS (dichlorphenamide) for patients with Primary Periodic Paralysis…
Even though a drug is sometimes developed with a certain condition in mind, it can be used to effectively treat or manage alternate conditions. For example, pembrolizumab (KEYTRUDA) is…
According to a July 13 news release, biopharmaceutical company Kedrion Biopharma ("Kedrion") recently treated its first pediatric patient within the Phase 3 KIDCARES10 clinical trial. Within the trial, Kedrion…
According to MedPage Today, new research suggests that adult obesity could increase the risk of developing Crohn's disease. Within the study, researchers evaluated how body mass index (BMI) impacted the…
Diet and nutrition play immense roles in our health. According to the Atlanta Journal-Constitution, for example, researchers discovered an association between colorectal cancer and eating red meat. “Hasn’t that been…
On July 8, 2021, gene therapy company Lysogene shared that its gene therapy candidate LYS-GM101, for patients with GM1 gangliosidosis, received Fast Track designation from the FDA. Currently, no treatments…
The ESMO World Congress on Gastrointestinal Cancer 2021 took place from June 30-July 3, 2021. During the Congress, researchers discussed data and research in the gastrointestinal field. According to MedScape,…
Towards the end of June 2021, biopharmaceutical company AbbVie shared that Upadacitinib (RINVOQ) reached both its primary and all secondary endpoints in a Phase 3 maintenance study evaluating the therapy…
ANGEL AID CARES began as the brainchild of Cristol Barrett O’Loughlin, out of a shared desire to pay tribute to her own family experience and to uplift others in need.…
Thus far, retifanlimab, a drug designed to treat patients with anal cancer, has received Orphan Drug designation and Priority Review status. However, the approval process has not been entirely easy.…
Have you ever heard of newborn screening? Basically, newborn screening is a public health service which can identify potential hormone-related, genetic, or metabolic conditions. Since early identification is so crucial…
Do you love golfing? Interested in making a difference for patients with amyotrophic lateral sclerosis (ALS)? If you're located in or around Oceanside, CA, then I have an event…
The FDA has a series of designations it grants to drugs or biologics intending to treat rare, serious, or life-threatening conditions. One of these is Breakthrough Therapy designation. According to…
Unfortunately, clinical trials do not always go the way that researchers intend. Sometimes, issues arise during early or preclinical studies which cause enough concern to stop or pause the…
According to MPR, the FDA recently granted Orphan Drug designation to CPI-613 (devimistat), an investigational treatment designed for patients with biliary cancer. Altogether, "biliary cancer" can consist of bile…
In the past, some drug developers were not incentivized to develop treatments for those within the rare disease community, considering the smaller patient size. However, the FDA created the Orphan…
Want to surgically remove a pancreatic tumor? Well, the entire process can take anywhere from 6-12 hours and presents some difficulties for medical practitioners. For example, during surgery, it can…
Often misdiagnosed as other conditions such as Rett syndrome or cerebral palsy, DDX3X mutations cause both cognitive and physical difficulties. Since only an estimated 700 patients have been diagnosed…
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