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September 19, 2019September 19, 2019

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

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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 ... Read more

September 3, 2019September 3, 2019

Lack of SIRT1 Likely Contributes to Neuroinflammation in X-ALD, Study Says

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Lack of a protein called sirtuin 1 (SIRT1) in cells that build up the lining of small blood vessels in the brain likely contributes to neuroinflammation associated with ... Read more

August 30, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 27, 2019August 27, 2019

Off-Label MS Therapy Rituximab Fails to Stop Progression of Advanced Childhood Cerebral ALD, Case Report Finds

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Off-label use of the multiple sclerosis (MS) therapy rituximab (sold as Rituxan in the U.S. and MabThera in Europe) may not be recommended to treat patients with advanced stage ... Read more

August 20, 2019August 20, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 13, 2019August 13, 2019

Blood Stem Cell Transplant Feasible, but Better Before Onset of Neurological Complications, Case Report Shows

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While allogeneic hematopoietic stem cell transplant (HSCT) is a feasible approach for people with adrenoleukodystrophy (ALD), it should be performed before the onset of neurological manifestations to effectively stop disease ... Read more

August 6, 2019August 6, 2019

First Clinical Trial of VK0214 to Treat X-linked ALD Planned for Early 2020, Viking Says

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Viking Therapeutics plans to launch a first clinical trial for VK0214, its investigational oral small molecule to treat adrenoleukodystrophy in 2020, the company announced. Adrenoleukodystrophy (ALD), also known as X-linked ALD (X-ALD), is caused ... Read more

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

August 1, 2019August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

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Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more

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