First Gene Therapy for Children With Metachromatic Leukodystrophy Approved by FDA
FRIDAY, March 22, 2024 -- The U.S. Food and Drug Administration has approved Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy (MLD).
Lenmeldy is a one-time, individualized... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - March 22, 2024 Category: Pharmaceuticals Source Type: news
The World ’s Most Expensive Drug Is Now a $4.25 Million Gene Therapy
A new gene therapy for an ultra-rare disease will have a wholesale cost of $4.25 million, making it the world’s most expensive drug.
The one-time treatment, Lenmeldy, won U.S. regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD.
MLD is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics said the drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday.
[time-brightcove not-tgx=”true”]
Read Mo...
Source: TIME: Health - March 21, 2024 Category: Consumer Health News Authors: Gerry Smith/Bloomberg Tags: Uncategorized healthscienceclimate wire Source Type: news
FDA OKs Gene Therapy for Metachromatic Leukodystrophy FDA OKs Gene Therapy for Metachromatic Leukodystrophy
Atidarsagene autotemcel (Lenmeldy) is approved for presymptomatic late infantile, presymptomatic early juvenile, or early symptomatic early juvenile MLD.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 19, 2024 Category: Neurology Tags: Neurology & Neurosurgery Source Type: news
Pediatric Neuron Disease Gets Its First Gene Therapy
(MedPage Today) -- The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - March 19, 2024 Category: American Health Source Type: news
FDA approves Orchard ’s gene therapy for children with rare genetic disease
This decision marks the first FDA-approved treatment for children with a rare genetic disease called metachromatic leukodystrophy (MLD) and Orchard's entry into the U.S. drug market. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 18, 2024 Category: Pharmaceuticals Authors: Hannah Green Source Type: news
FDA Approves Lenmeldy (atidarsagene autotemcel) for Children with Metachromatic Leukodystrophy
TOKYO and LONDON and BOSTON, March 18, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the U.S. Food and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - March 18, 2024 Category: Drugs & Pharmacology Source Type: news
Mother's agony as three-year-old daughter's squint turns out to be a deadly brain disorder that kills most kids before they turn five
Nellie (pictured), from Brighton, was diagnosed with metachromatic leukodystrophy - a rare genetic disease causing severe damage to the nervous system and organs. (Source: the Mail online | Health)
Source: the Mail online | Health - October 17, 2023 Category: Consumer Health News Source Type: news
Girl with deadly inherited condition is cured with gene therapy on NHS
Teddi Shaw, from Northumberland, first recipient on health service of Libmeldy, world ’s most expensive drugA girl born with a rare and deadly genetic condition is expected to live a long and normal life after becoming the first person to be cured on the NHS with the help of a revolutionary gene therapy.Teddi Shaw was diagnosed with metachromatic leukodystrophy (MLD), an inherited condition that causes catastrophic damage to the nervous system and organs. Those affected usually die young.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - February 15, 2023 Category: Science Authors: Andrew Gregory Health editor Tags: NHS Health Society Northumberland UK news Source Type: news
Baby suffering from rare condition is saved by the world’s most expensive drug
Teddi Shaw receives a gene therapy treatment for rare deadly genetic condition called metachromatic leukodystrophy . (Source: Daily Express - Health)
Source: Daily Express - Health - February 14, 2023 Category: Consumer Health News Source Type: news
2022 Health Care Heroes: Dr. Paul Orchard develops life-saving gene therapy treatment
Dr. Paul Orchard made history Sept. 27, 2021, when his pediatric patient became the first person in the United States to receive life-saving gene therapy treatment for metachromatic leukodystrophy, a rare, progressive and generally fatal genetic disease. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 17, 2022 Category: Biotechnology Authors: Brigitt Martin Source Type: news
'Absolutely heartbreaking': Mother's agony as daughter battles deadly disease - symptoms
A MOTHER in desperation is hoping that potentially life-saving treatment will be developed in time to save her 13-year-old daughter Aggie, who suffers from a severe yet rare form of TUBB4a-related leukodystrophy - a genetic disease that affects the central nervous system. (Source: Daily Express - Health)
Source: Daily Express - Health - May 22, 2022 Category: Consumer Health News Source Type: news
Tens of thousands could have 'incurable' disorder 'similar' to cerebral palsy - symptoms
HEALTH experts have warned that thousands could unknowingly suffer from a rare life-threatening disease. Although leukodystrophy has long been considered a rarity, researchers believe the number of people affected may be significantly higher than previously thought. Experts have now shared the key warning signs of the condition. (Source: Daily Express - Health)
Source: Daily Express - Health - February 9, 2022 Category: Consumer Health News Source Type: news
Toddler with frequent falls and neuroregression: imaging clues! - Sharawat IK, Dawman L, Panda PK.
Late infantile metachromatic leukodystrophy is an autosomal recessive disorder caused by a deficiency in the enzyme activity of Aryl sulfatase-A. The classical presentation is characterized by gait disturbance, frequent fall, toe walking, impaired swallowi... (Source: SafetyLit)
Source: SafetyLit - February 6, 2021 Category: International Medicine & Public Health Tags: Age: Infants and Children Source Type: news
How the ketogenic diet helped 5-year-old control his seizures
When Rebecca and Stuart Galbreath brought their twin boys Jake and Brody home, they quickly realized something wasn't quite right.
"When we'd go to take the twins' picture, Brody would look at the camera, but Jake would be looking off," recalls Rebecca. At first, they thought he might be blind. But he was also crying more than other babies and never slept.
An MRI prescribed by his neurologist at Atrium Health Cabarrus pinpoi nted the issue: Jake was suffering from leukodystrophy, a rare type of… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 1, 2019 Category: Pharmaceuticals Authors: Atrium Health Source Type: news
How the ketogenic diet helped 5-year-old control his seizures
When Rebecca and Stuart Galbreath brought their twin boys Jake and Brody home, they quickly realized something wasn't quite right.
"When we'd go to take the twins' picture, Brody would look at the camera, but Jake would be looking off," recalls Rebecca. At first, they thought he might be blind. But he was also crying more than other babies and never slept.
An MRI prescribed by his neurologist at Atrium Health Cabarrus pinpoi nted the issue: Jake was suffering from leukodystrophy, a rare type of… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 1, 2019 Category: Biotechnology Authors: Atrium Health Source Type: news
