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 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…
When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting…
Near the end of September, Hansa Biopharma ("Hansa") announced promising data from a Phase 2 clinical trial on imlifidase. This therapy is designed to treat patients with severe anti-GBM antibody…
For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…
If you're like me, you might be surprised that COVID-19 is not only still deeply ingrained in our world, but it seems to continue growing. As of the afternoon of…
At the start of October, Swedish-based IRLAB announced that the FDA approved an investigational new drug application (IND) for their therapeutic candidate mesdopetam (IRL790). This therapy is designed to treat…
According to a press release, biopharmaceutical company Apellis Pharmaceuticals ("Apellis") recently launched registration programs to explore pegcetacoplan for patients with C3 glomerulopathy (C3G), amyotrophic lateral sclerosis (ALS), and immune…
At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
For patients with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), post-exertional malaise (PEM) can be extremely debilitating and disruptive. PEM occurs when symptoms worsen or become more severe within 12-48 hours…
In late September, GlaxoSmithKline announced FDA approval of its treatment, Nucala, for patients with hypereosinophilic syndrome (HES). The treatment is safe for patients older than 12. Additionally, this approval is…
On September 30, 2020, BioCryst Pharmaceuticals ("BioCryst") announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously…
Historically, clinical trials have played an immense role in collecting patient data, understanding disease progression, and crafting more targeted treatments. This is especially true in the oncology realm. But according…
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