According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as…
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 a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…
Robert is a 43 year old father of a two year old son named Parker. Robert was diagnosed with inoperable stage 4 bladder cancer about a year and a half…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
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…
The annual meeting of the American College of Rheumatology was recently held, where data was presented on Cimzia and Bimekizumab. Not only were both of these therapies found to benefit…
"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…
According to Biospace, Acceleron Pharma has presented preliminary data from their ongoing Phase 2 trial of sotatercept, a treatment for pulmonary arterial hypertension (PAH). The study, titled SPECTRA, is designed…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a story from GlobeNewswire, the rare disease liver company Albireo Pharma, Inc., recently announced positive results from a phase 3 study testing the company's experimental drug odevixibat as…
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…
According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…
Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…
According to a story from Charcot-Marie-Tooth News, patients living with Charcot-Marie-Tooth disease have reported in a recent survey that their symptoms of pain and decline in mobility increased during the…
Syndax Pharmaceuticals has plans to release data on axatilimab, a treatment for chronic graft-versus-host disease (cGVHD), at the 62nd American Society of Hematology (ASH) Annual Meeting, which will take place…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy, or SMA, is a genetic disease that affects an individual’s central and peripheral nervous systems, as well as their…
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…
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