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 October 22, 2020, BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from a Phase 3 clinical trial evaluating berotralstat for patients with hereditary angioedema (HAE). As a…
In early October, the FDA approved an Investigational New Drug Application (IND) for APR-548, a potential treatment for patients with TP53 mutant myelodysplastic syndromes (MDS). According to a press release from…
In mid-October, biopharmaceutical company Zogenix announced that its Dravet syndrome treatment FINTEPLA (fenfluramine) received a positive CHMP opinion. The CHMP is part of the European Medicines Agency (EMA). While…
Earlier this week, the FDA granted Priority Review status to use a combination of Opdivo-Cabometyx as a treatment for patients with renal cell carcinoma (RCC). According to Healio, Opdivo (nivolumab)…
Many people within the rare disease community share a common experience: feeling isolated or misunderstood because of your condition. In some cases, such as conditions causing chronic pain or fatigue,…
At the very core of genetic diseases are - well, our genes. However, each year, an estimated 400,000 infants are born with de novo mutations, or new and spontaneous gene mutations not…
When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…
According to Biotech365, the National Hemophilia Foundation (NHF) recently revised its treatment recommendations for patients with congenital fibrinogen deficiency, or Factor I deficiency. Now, intravenously administered fibryga, developed by…
Recently, clinical-stage biopharmaceutical company Imara Inc. ("Imara") announced that the first patient was dosed in the Phase 2b Forte clinical trial. The trial is evaluating IMR-687, a PDE9-inhibitor, for…
Later this week, from October 21-23, presenters and attendees will be present at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Although this year is slightly different, considering the…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
What are the best treatments and diagnostic approaches for blood disorders like aplastic anemia and myelodysplastic syndromes (MDS)? The UT Southwestern Medical Center is trying to streamline these options and…
According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical…
On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat…
Currently, most treatments for scleroderma, a group of conditions causing the hardening of skin and connective tissue, are symptomatic. That is, they treat symptoms but not the underlying cause of…
In December 2019, the FDA approved Ervebo, the first-ever Ebola vaccine. However, despite preventative measures, there still was never any actually approved treatment options - until now. Recently, the FDA…
Recently, during the virtual 28th United European Gastroenterology Week Congress, the Janssen Pharmaceutical Companies of Johnson & Johnson announced 5-year results from a Phase 3 extension study on STELARA…
In early October, global biopharmaceutical company Bristol Myers Squibb announced the Canadian approval of ZEPOSIA (ozanimod) for patients with multiple sclerosis (MS). In particular, the drug is designed to…
Over this past week, the Harrington Discovery Institute announced five recipients of its introductory Harrington UK Rare Disease Scholar Award competition. The Institute, which is involved with Cleveland's University Hospitals,…
About one month ago, the HealthWell Foundation announced the beginning of the Adrenal Insufficiency Fund. Designed to assist patients with adrenal insufficiency, the fund will grant financial assistance to…
While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…
In recent years, there have been huge progressions in the field of gene therapy. At its core, gene therapy addresses genetic disorders by using genes to directly treat or…
Last week, clinical stage biopharmaceutical company Neurotrope announced that the first patient was dosed in a Phase 2 clinical trial to test the safety, efficacy, and tolerability of bryostatin-1…
In the past, there existed no approved treatments for alkaptonuria (AKU), a rare genetic disorder characterized by homogentisate dioxygenase deficiency. However, according to Medical XPress, this recently changed as British…
In conditions like multiple sclerosis or neuromyelitis optica, disease progression is spurred by the destruction of myelin, the protective coating around nerve cells. However, new research suggests that N-acetylglucosamine, a…
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