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…
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…
Before you read on, make sure to check out Part 1 of our interview. In Part 1, Chris Peetz discusses his background, why he joined Mirum, and the process of evaluating LIVMARLI…
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…
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…
The 9th International Symposium on Alagille Syndrome "The Alagille Trail" July 16-17, 2021 The Alagille Syndrome Alliance is Discovering New Paths and Leaving No Stone Unturned at their 9th annual…
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,…
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,…
The International Symposium 2019 "Connecting The Pieces" Cincinnati, Ohio This will be the 8th International Symposium hosted by the Alagille Syndrome Alliance. This meeting is the largest gathering in the…
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 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…
According to a story from BioSpace, the orphan liver disease drug company Albireo Pharma recently announced that its investigational product A4250 was granted Orphan Drug designation by the US Food…
The Alagille Syndrome Alliance has launched its first ever awareness ribbon in support of the Alagille syndrome (ALGS) community! The multi-colored awareness ribbon is meant to represent the complex and uniquely…
The Gala of Dreams "The Dawn of a Dream" This is the first gala event hosted by the Alagille Syndrome Alliance (ALGSA). Open to all members of the Alagille syndrome…
PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
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