For as long as she can remember, eighth-grader Ella Bork has held a keen knowledge of rare diseases, especially Alagille syndrome. Her mother, Cher, is actually the Executive Director of…
Have you ever had an itch that you just can't seem to scratch? Intensely itchy skin, also known as pruritus, is a hallmark characteristic of many conditions that affect the…
In many rare conditions, early diagnosis and treatment typically correspond with better outcomes. Prior to March 2023, LIVMARLI (maralixibat) was approved for the treatment of cholestatic pruritus related to Alagille…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Treatment for Alagille syndrome, a rare genetic disorder that affects multiple organ systems, are typically symptomatic. Many children born with this disorder eventually require a liver transplant; without this, Alagille…
Each week, in an email to his team, Albireo Pharma CEO Ron Cooper sends out the story of a child or family affected by conditions such as PFIC or Alagille…
The American Association for the Study of Liver Disease (AASLD) held its annual Liver Meeting from November 4-7, 2022 in Washington, D.C. During the meeting, Mirum Pharmaceuticals shared two late-breaker…
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…
CANbridge Pharmaceuticals recently announced via press release that the Taiwan Food and Drug Administration (TFDA) has accepted their New Drug Application (NDA)/Orphan Drug Registration (ODR) for CAN108. Marketed as LIVMARLI,…
In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However,…
When Karter was first born 14 months ago, his mother Breonna was overjoyed. But she quickly realized a series of upcoming obstacles when, at birth, Karter was diagnosed with Alagille…
In a recent press release, biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum") shared that the company finished its rolling New Drug Application (NDA) submission. The NDA focuses around maralixibat, an investigational…
Each year, January 24 marks International Alagille Syndrome Awareness Day. During this day, organizations across the globe raise awareness around Alagille Syndrome (ALGS), from offering educational resources to allowing patients…
According to a press release from the rare liver disease company Albireo Pharma, Inc., the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for odevixibat.…
Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo's mission,…
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…
Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16.…
This year, the Digital International Liver Congress took place online from August 27-29, thanks to COVID-19. During the conference, biopharmaceutical company Mirum Pharmaceuticals announced data from its Phase 2 INDIGO…
By Danielle Bradshaw from In The Cloud Copy Sean and Josie Kelly, a New York native couple, have had a newborn son named Sawyer who was born with a rare…
International Alagille Awareness Day The very first International Alagille Awareness day was held on January 24th, 2020. What is Alagille syndrome? It is a rare disease which effects the heart,…
According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…
According to a story from abc13.com, Makenna Brown, a four-year-old girl from Texas, has been plagued by relentless itching thanks to her Alagille syndrome, a rare genetic disorder. Her distressed…
According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…
According to a story from prnewswire.com, the drug developer Mirum Pharmaceuticals recently announced that is has entered an agreement with Shire which grants exclusive rights for marketing and development of…
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