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Zilganersen Gains FDA Fast Track for Alexander Disease, a Rare Leukodystrophy
09 Oct 2024 19:08 GMT
… and ultimately fatal type of leukodystrophy. Otherwise known as ION373, the …
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7's HERO: 6-year-old boy with leukodystrophy is the Boise State Broncos' newest and youngest recruit
09 Sep 2024 04:42 GMT
… has a rare form of leukodystrophy. It impacts his brain and … a rare form of 4H leukodystrophy. The diagnosis was heartbreaking.
…
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Release snags seed funding for metachromatic leukodystrophy research
06 Aug 2024 20:04 GMT
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Tocilizumab May Be Effective Option for Leukodystrophy-Like Pattern Forms of MOGAD
22 Jul 2024 22:57 GMT
… with white matter lesions mimicking leukodystrophy, showed improvement in cognitive … differential diagnosis between MOGAD and leukodystrophy could be challenging. The … usually preserved in patients with leukodystrophies."
Tocilizumab was also …
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Navigating a Novel Therapy Frontier: Gene Therapy's Stunted Impact on Leukodystrophies
01 Jul 2024 21:46 GMT
… including adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD), Krabbe disease or … for Children with Metachromatic Leukodystrophy. News release. Orchard Therapeutics … with Early-onset Metachromatic Leukodystrophy. News release. Orchard Therapeutics …
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Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy
30 May 2024 16:39 GMT
… patients with early-onset metachromatic leukodystrophy (MLD). Long-term data from … and Motor Development in Metachromatic Leukodystrophy with up to 12 Years … Therapy for Children with Metachromatic Leukodystrophy. News release. Orchard Therapeutics. March …
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From Research To Reality: Gene Therapy For Metachromatic Leukodystrophy
07 May 2024 18:12 GMT
… caused by genetic factors. Metachromatic leukodystrophy, MLD for short, is a … MLD.
What is Metachromatic Leukodystrophy?
Metachromatic leukodystrophy is a rare genetic disorder …
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Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy
20 Mar 2024 11:00 GMT
… early juvenile (ESEJ) metachromatic leukodystrophy (MLD). For additional details … symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). IMPORTANT SAFETY INFORMATION …
iMahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with …
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US FDA approves Orchard Therapeutics' Lenmeldy for children with early─onset metachromatic leukodystrophy
20 Mar 2024 10:45 GMT
… to as early-onset—metachromatic leukodystrophy (MLD).
“The FDA approval … symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
In Europe, Lenmeldy … symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
Orchard Therapeutics, a …
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FDA approves Orchard’s Lenmeldy gene therapy for metachromatic leukodystrophy
20 Mar 2024 12:45 GMT
… early symptomatic early juvenile metachromatic leukodystrophy (MLD).
The approval marks the …
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