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…
by Danielle Bradshaw from In The Cloud Copy A recently performed study has demonstrated that it is possible to use tablets to screen patients that have chronic lung conditions -…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
by Lauren Taylor from In The Cloud Copy Nonalcoholic fatty liver disease, or NAFLD, and nonalcoholic steatohepatitis, or NASH, are two conditions affecting the liver that plague millions of Americans…
Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…
IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…
As reported in the BBC; Aaron Bradley is concerned that returning to work will endanger his son’s health. This follows the Scottish governments decision to move his toddler off of…
Sierra Oncology has recently released two abstracts that will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting. The meeting is planned for December 5-8. Both abstracts…
According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test…
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…
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