Posts by: Marisa Wexler

News

Treatment with Minoryx Therapeutics’ leriglitazone in a Phase 2/3 clinical trial was found to improve motor function in people with early symptomatic adrenomyeloneuropathy (AMN), one of the most common types of ... Read more

November 4, 2020November 4, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations ... Read more

October 21, 2020October 21, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series ... Read more

May 12, 2020May 12, 2020

New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

News

Hitachi Chemical Advanced Therapeutics Solutions and Apceth Biopharma GmbH, both subsidiaries of Hitachi Chemical Company, recently signed a new long-term development and manufacturing agreement with Bluebird Bio. The agreement will expand ... Read more

April 28, 2020April 28, 2020

Case Report Describes Novel ALD-causing Gene Mutation

News

A new adrenoleukodystrophy-causing mutation in the ABCD1 gene was described in the case report of a 20-year-old Korean man. The report, “Novel ABCD1 Gene Mutation in a Korean Patient with X-Linked Adrenoleukodystrophy ... Read more

September 19, 2019September 19, 2019

Clinical Trial Data Support Safety, Efficacy of Lenti-D for Boys with CALD

News

New Phase 2/3 clinical trial data suggests that the gene therapy candidate Lenti-D is safe and effective for the treatment of cerebral adrenoleukodystrophy (CALD), Bluebird Bio announced. The most ... Read more

July 23, 2019July 23, 2019

Autism and ADHD Possible Early Signs of ALD, According to Case Report

News

The case of a 19-year-old patient diagnosed with adrenomyeloneuropathy (AMN), the adult-onset form of adrenoleukodystrophy (ALD), highlights how attention and behavioral issues may be early indicators of ALD. Findings were ... Read more

July 9, 2019July 9, 2019

Report of Siblings with X-ALD Highlights Different Disease Manifestations

News

A new case report describes two siblings with X-linked adrenoleukodystrophy (X-ALD) who had very different manifestations of the disease. The study, “A Distinct Clinical Phenotype in Two Siblings with X-linked ... Read more