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.
On June 22, 2021, PharmaTimes Online reported that the European Commission (EC) granted conditional approval to Koselugo (selumetinib), an orally administered therapy, for use in pediatric patients with neurofibromatosis…
During the European Hematology Association (EHA) Annual Congress (EHA2021), which took place virtually this year, hematological experts and stakeholders discussed new advances in the field. According to Fierce Biotech, one…
In early June 2021, Epidermolysis Bullosa News reported that Filsuvez (Oleogel-S10), a topical gel for epidermolysis bullosa (EB), received Priority Review designation from the FDA. Filsuvez Developed by Amryt Pharma,…
In a news release from June 23, 2021, Revolo Biotherapeutics ("Revolo") shared that the FDA approved an Investigational New Drug (IND) application for the company's '1104 peptide treatment. Altogether, '1104…
Orphan Drug designation is a status granted to drugs or biologics intended to treat patients with rare conditions, defined as those affecting under 200,000 Americans. According to Pulmonary Hypertension News,…
Mal de débarquement, or "sickness of disembarkment," is a phenomenon which causes a bobbing, rocking, or swaying sensation, alongside other symptoms. With many people, mal de débarquement only lasts…
Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV), or alveolar capillary dysplasia (ACD) for short, is a rare, congenital, and life-threatening disorder. Without receiving lung transplants in infancy,…
Transcription factor SOX 11 is encoded by the SOX11 gene. Altogether, this protein is known to play a role in embryonic neurogenesis and tissue formation. However, an estimated 90% of patients with…
When Taylor Brown was just two years old, her mother TaMetress Reed began noticing certain symptoms, such as hardening and tightening patches of skin. As Brown grew up, the symptoms…
According to a relatively recent news release from biopharmaceutical company Applied Therapeutics, Inc., AT-007 received Fast Track designation from the FDA. This therapy, which has also received both Orphan Drug…
Internationally, over 400,000 people die of malaria each year. In many cases, a majority of these deaths occur in sub-Saharan Africa; children are the most vulnerable. However, according to Medical…
For the first time, a research study has discovered a genetic marker related to high-risk neuroblastoma. According to Medical XPress, researchers determined that anaplastic lymphoma kinase (ALK) gene mutations and…
As reported by Oncology Nursing News, gunagratinib (ICP-192) was recently granted Orphan Drug designation by the FDA. Altogether, this treatment is being evaluated as a potential treatment for patients…
Currently, there are no real treatments for patients with primary mitochondrial myopathy (PMM). Most therapies, such as physical, occupational, or speech therapy, are designed to simply lessen or mitigate symptoms.…
In late May 2021, the FDA granted accelerated approval to Truseltiq (infigratinib). As reported by Cure, this therapy is designed for adult patients with unresectable locally advanced, previously treated, or…
When Stephanie Ernst-Milner first discovered that she was having twins, she was speechless. But what she learned over the course of her pregnancy, and in the seven years since, has…
Mobility issues are common in patients with osteogenesis imperfecta (OI) - especially tendinopathy, or tendonitis. But why does this happen? According to Medical XPress, a study performed by Baylor College…
For years, researchers have been evaluating pridopidine, developed by Prilenia Therapeutics ("Prilenia"), as a potential therapy for patients with neurodegenerative conditions. As reported in HD Buzz, new research has highlighted…
Although it is still considered an experimental technique, gene therapy shows promise for treating a wide variety of genetic diseases or disorders. In a recent news release from the Sanford…
Over the past few years, Elmiron (pentosan polysulfate sodium, "PPS"), a therapy designed to treat patients with interstitial cystitis (IC), has been associated with potential vision damage. Despite a variety…
From June 5-8, 2021, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Virtual Meeting. Due to COVID-19, the meeting was held fully online. However, that…
Some conditions or diseases are more prevalent in certain groups. In the case of idiopathic intracranial hypertension (IIH), new research suggests that it is much more common in Black or…
Normally, cerebrospinal fluid (CSF) surrounds our brain and spinal cord, providing numerous benefits from injury protection and nutrient delivery to waste removal. Hydrocephalus, or "water on the brain," occurs when…
In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. ("Timber") shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial,…
Could a specialized cancer vaccine actually improve patient outcomes? According to a news release from biotechnology company AIVITA Biomedical, Inc. ("AIVITA"), the answer could be "yes." Citing data from a…
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