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.
The 62nd American Society of Hematology (ASH) Annual Meeting and Exposition will be held virtually this year from December 5 through 8. The goal of the meeting is to provide…
In a recent press release, gene therapy company Orchard Therapeutics ("Orchard") announced that the FDA approved Orchard's Investigational New Drug (IND) application for OTL-200. This gene therapy solution is…
Recently, the American College of Rheumatology (ACR) virtually held ACR Convergence 2020, the organization's annual meeting. At the meeting, researchers shared new insights in the realm of rheumatology; these were…
From November 13-16, researchers, doctors, and others participated in the American Association for the Study of Liver Diseases (AASLD)'s Annual Meeting, or The Liver Meeting Digital Experience. During the…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
Earlier this month, biopharmaceutical company Applied Molecular Transport (AMT) announced the publication of its preclinical data in the Journal of Immunology. You can find the published article here. Within the study,…
Recently, biopharmaceutical company Oncternal Therapeutics ("Oncternal") announced that it has additional clinical data available from its Phase 1 clinical trial. Through the trial, researchers are analyzing an investigational therapy, TK216,…
During the Association for the Study of Liver Diseases (AASLD) Liver Meeting, which took place from November 13 to 16, biopharmaceutical company Albireo Pharma ("Albireo) announced data from the Phase…
According to Charcot-Marie-Tooth News, biotechnology company AcuraStem recently identified 37 potentially therapeutic compounds that could be used to treat patients with Charcot-Marie-Tooth disease type 2A (CMT2A). Currently, CMT2A is the…
"Repurposing" drugs means that researchers may discover that certain compounds or therapies for one condition could be translated into the treatment plan for other conditions. Recently, research published in the American…
While there are some treatment options for Acanthamoeba keratitis (AK), they often vary from country to country, or even doctor to doctor. The variability could worsen patient outcomes. As a result, a…
From November 13th through the 15th, the American College of Allergy, Asthma & Immunology (ACAAI) held their 2020 Annual Scientific Meeting. While the coronavirus pandemic necessitated that the meeting be…
While gene therapy offers the promise of addressing and treating many genetic disorders, creating a better quality of life for many people, there are also ethical issues associated with…
Although the American College of Asthma, Allergy and Immunology (ACAAI) Annual Scientific Meeting has wrapped up for this year, there is still a ton to learn from the presentations…
This year, the American College of Rheumatology (ACR) held their Convergence 2020 meeting virtually from November 5-9, with additional poster presentations on November 10 and 21. According to a…
In mid-November, The Assistance Fund (TAF) announced that they were beginning a copay assistance program for those with acromegaly. For nearly 11 years, TAF has worked to assist patients…
Recently, biopharmaceutical company Nkarta announced the dosing of the first patient in its Phase 1 clinical trial. Within the trial, researchers will evaluate and analyze the company's investigational therapy,…
In mid-November, 2020, Rocket Pharmaceuticals announced that the company received a $3.7M grant from the California Institute for Regenerative Medicine to study potential treatments for malignant infantile osteopetrosis. In…
Early last week, Agios Pharmaceuticals ("Agios") announced that its treatment Mitapivat received Orphan Drug designation. This orally administered therapy is a pyruvate kinase R (PKR) activator. As Agios has…
From November 5 through 9th, the American College of Rheumatology (ACR) held their ACR Convergence 2020. Though the annual meeting took place online, it was still rife with insights into…
Did you know that there are multiple forms of multiple sclerosis (MS)? The most common form of MS is relapsing-remitting, meaning patients go through symptomatic periods and periods of remission.…
In late October, the American Society of Nephrology (ASN) held the ASN Kidney Week 2020 Reimagined. During the conference, biopharmaceutical company FibroGen shared two posters (abstracts PO2625 and PO2626) regarding…
As many patients with rare diseases know, it can sometimes be difficult to achieve the correct diagnosis. In fact, the average length of time it takes from symptom onset to…
In a recent press release, biopharmaceutical company Rallybio announced that the first participant was dosed in a Phase 1/2 clinical trial. Currently, only healthy male participants are enrolled in the…
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