A majority of available treatment options for metachromatic leukodystrophy (MLD) rely on relieving symptoms. However, the recent approval of Lenmeldy (atidarsagene autotemcel) is the first ever FDA-approved gene therapy option…
Patient Worthy partner Alex TLC has announced that, in a large-scale newborn screening study supported by the UK's NHS and Genomics England, adrenoleukodystrophy (ALD) and metachromatic leukodystrophy (MLD) will be…
Within the first few months after Reuben Jayce-Mills was born, he seemed to be progressing fairly normally. But when Reuben turned nine months old, his parents noticed some concerning…
Ally and Jake Shaw always dreamed of having a big, happy family. The pair began their journey with their daughter Nala, now just under 3 years old, and later brought…
Celia Grace, a metachromatic leukodystrophy (MLD) patient from Alabama, has recently made history in Minnesota - and she's just five years old! Celia recently underwent an investigational gene therapy to…
Dave and Kendra Riley have a beautiful family: them and their daughters Eva, Kiera, and Olivia. Yet the family has faced some scary and difficult obstacles over the last…
Victoria and Zack Rasberry are asking for a miracle to save their youngest child, Ollie. Ollie and his older sister, Addy, both have metachromatic leukodystrophy (MLD), a rare disorder that…
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…
As reported in Biospace, the National Organization for Rare Disorders (NORD) has just opened registration for patients to take part in a natural history study on metachromatic leukodystrophy (MLD), a…
By: Natalie Homan from In The Cloud Copy Connie Elson was a happy, independent little girl before she started showing worrying symptoms at the age of four. After many doctor…
In a post on the Metachromatic Leukodystrophy (MLD) Foundation's website, the organization discusses the possibilities of using gene therapy as a treatment for this rare genetic disorder. The page discusses…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
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A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
The development of treatments and medicines for diseases can take a long time. These treatments come from many hours of research, studies, trials, and waiting for approval from the…
According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…
According to a story from EPM Magazine, the pharmaceutical company Orchard Therapeutics received Rare Pediatric Disease Designation for its gene therapy drug candidate OTL-200. The therapy is in development for…
During a charity event, a proud UK father pushed his daughter on her wheelchair for 21 miles to raise money for her rare disease. Connie Elson is only 9-years-old, but…
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