What that can cost: ASPIRO, a gene therapy trial in boys under five with X-linked myotubular myopathy, tested two dose levels. Seven boys got 1.3 × 10¹⁴ vector genomes per kilogram. Seventeen got 3.5 × 10¹⁴, about 2.7 times higher. (5/8)
October 27, 2026 at 12:33 AM
What that can cost: ASPIRO, a gene therapy trial in boys under five with X-linked myotubular myopathy, tested two dose levels. Seven boys got 1.3 × 10¹⁴ vector genomes per kilogram. Seventeen got 3.5 × 10¹⁴, about 2.7 times higher. (5/8)
Cross-species transcriptomic atlas of X-linked Myotubular Myopathy identifies unique, treatment responsive myofiber populations https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
October 19, 2026 at 9:30 PM
Cross-species transcriptomic atlas of X-linked Myotubular Myopathy identifies unique, treatment responsive myofiber populations https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
Cross-species transcriptomic atlas of X-linked Myotubular Myopathy identifies unique, treatment responsive myofiber populations https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
October 19, 2026 at 9:30 PM
Cross-species transcriptomic atlas of X-linked Myotubular Myopathy identifies unique, treatment responsive myofiber populations https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
Cross-species transcriptomic atlas of X-linked Myotubular Myopathy identifies unique, treatment responsive myofiber populations #SingleCell 🧪🧬🖥️
https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
October 19, 2026 at 10:00 PM
Cross-species transcriptomic atlas of X-linked Myotubular Myopathy identifies unique, treatment responsive myofiber populations #SingleCell 🧪🧬🖥️
https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
https://www.biorxiv.org/content/10.64898/2026.10.16.759495v1
Speakers
• Dr. James Dowling (Chair): Clinical safety signals and efficacy of AAV gene therapy in neuromuscular disorders
• Dr. Emanuela Pannia: Preclinical models and mechanisms of AAV-associated hepatotoxicity: lessons learned from X-linked myotubular myopathy.
• Dr. James Dowling (Chair): Clinical safety signals and efficacy of AAV gene therapy in neuromuscular disorders
• Dr. Emanuela Pannia: Preclinical models and mechanisms of AAV-associated hepatotoxicity: lessons learned from X-linked myotubular myopathy.
October 12, 2026 at 12:02 PM
Speakers
• Dr. James Dowling (Chair): Clinical safety signals and efficacy of AAV gene therapy in neuromuscular disorders
• Dr. Emanuela Pannia: Preclinical models and mechanisms of AAV-associated hepatotoxicity: lessons learned from X-linked myotubular myopathy.
• Dr. James Dowling (Chair): Clinical safety signals and efficacy of AAV gene therapy in neuromuscular disorders
• Dr. Emanuela Pannia: Preclinical models and mechanisms of AAV-associated hepatotoxicity: lessons learned from X-linked myotubular myopathy.
Fatal cases of liver failures have been reported for X-linked myotubular myopathy (XLMTM), DMD, SMA and LGMD. When liver injury is observed, this is treated by immune suppression.
October 6, 2026 at 5:30 AM
Fatal cases of liver failures have been reported for X-linked myotubular myopathy (XLMTM), DMD, SMA and LGMD. When liver injury is observed, this is treated by immune suppression.
Please take a few minutes to watch this video by Jessica Hair. Her son, Declan, was born with a rare condition called MTM (Myotubular myopathy). She talks about how the loss of DEI, the Dept of Ed, IDEA, and Medicaid cuts will hurt her family.
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March 22, 2025 at 6:36 PM
Please take a few minutes to watch this video by Jessica Hair. Her son, Declan, was born with a rare condition called MTM (Myotubular myopathy). She talks about how the loss of DEI, the Dept of Ed, IDEA, and Medicaid cuts will hurt her family.
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Revealing a precise therapeutic target in X-linked centronuclear myopathy: doi.org/10.1172/jci....
Jocelyn Laporte & team perform in vivo studies of DNM2 functions in mice, identifing lipid binding as the principal driver of myotubular myopathy.
Jocelyn Laporte & team perform in vivo studies of DNM2 functions in mice, identifing lipid binding as the principal driver of myotubular myopathy.
May 21, 2026 at 11:03 AM
Revealing a precise therapeutic target in X-linked centronuclear myopathy: doi.org/10.1172/jci....
Jocelyn Laporte & team perform in vivo studies of DNM2 functions in mice, identifing lipid binding as the principal driver of myotubular myopathy.
Jocelyn Laporte & team perform in vivo studies of DNM2 functions in mice, identifing lipid binding as the principal driver of myotubular myopathy.
When to target the most abundant protein in the heart?
@droch.bsky.social & team show myosin becomes maladaptive in X-linked myotubular myopathy, consuming excessive energy, and inhibiting its ATPase activity in mice partly rescues the molecular defects: doi.org/10.1172/jci....
@droch.bsky.social & team show myosin becomes maladaptive in X-linked myotubular myopathy, consuming excessive energy, and inhibiting its ATPase activity in mice partly rescues the molecular defects: doi.org/10.1172/jci....
December 24, 2025 at 2:01 PM
When to target the most abundant protein in the heart?
@droch.bsky.social & team show myosin becomes maladaptive in X-linked myotubular myopathy, consuming excessive energy, and inhibiting its ATPase activity in mice partly rescues the molecular defects: doi.org/10.1172/jci....
@droch.bsky.social & team show myosin becomes maladaptive in X-linked myotubular myopathy, consuming excessive energy, and inhibiting its ATPase activity in mice partly rescues the molecular defects: doi.org/10.1172/jci....
Liver failure has been seen outside of Duchenne trials as well and is likely due to a T-cell response. In X-linked myotubular myopathy patients had a preexisting liver condition, which resulted in much higher incidence of liver problems and liver failure and death.
January 19, 2026 at 12:27 PM
Liver failure has been seen outside of Duchenne trials as well and is likely due to a T-cell response. In X-linked myotubular myopathy patients had a preexisting liver condition, which resulted in much higher incidence of liver problems and liver failure and death.
X-linked myotubular myopathy is a severe muscle disease, that primarily occurs in boys as the gene that is affected is located on the X-chromosome. This gene, the MTM1 gene, codes for a protein called myotubulin. Lacking this protein patients develop severe muscle weakness.
October 9, 2025 at 10:22 AM
X-linked myotubular myopathy is a severe muscle disease, that primarily occurs in boys as the gene that is affected is located on the X-chromosome. This gene, the MTM1 gene, codes for a protein called myotubulin. Lacking this protein patients develop severe muscle weakness.
#apaperaday Today the @worldmusclesociety.org #WMS2025 themed series continues with a pick from Shieh et al in Therapeutic Advances in Rare Diseases. It is a plain language summary of a previous publication on the gene therapy trial in myotubular myopathy patients. DOI 10.1177/26330040251362885
October 9, 2025 at 10:21 AM
#apaperaday Today the @worldmusclesociety.org #WMS2025 themed series continues with a pick from Shieh et al in Therapeutic Advances in Rare Diseases. It is a plain language summary of a previous publication on the gene therapy trial in myotubular myopathy patients. DOI 10.1177/26330040251362885
Authors do not mention the X-linked myotubular myopathy trial where patients died due to liver pathology that was present in patients before treatment but never recognized. Authors introduce and explain different approaches such as siRNA, ASO (RNase H and splice modulating) and gene editing.
July 16, 2025 at 7:16 AM
Authors do not mention the X-linked myotubular myopathy trial where patients died due to liver pathology that was present in patients before treatment but never recognized. Authors introduce and explain different approaches such as siRNA, ASO (RNase H and splice modulating) and gene editing.
Diet and gene therapy together may affect liver health in a rare muscle condition called X-linked myotubular myopathy (XLMTM). bit.ly/3NVBKB5
Diet and gene therapy together may affect liver health in a rare muscle condition
Research finds nutrition, disease biology and gene therapy delivery can interact to affect liver health in children with X-linked myotubular myopathy, offering insights for future gene therapy…
bit.ly
March 23, 2026 at 1:25 PM
Diet and gene therapy together may affect liver health in a rare muscle condition called X-linked myotubular myopathy (XLMTM). bit.ly/3NVBKB5
X #LINKED #MYOTUBULAR #MYOPATHY
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April 17, 2026 at 8:52 PM
Scientists Have Discovered a Simple Supplement That Causes Prostate Cancer Cells To Self-Destruct
Scientists Have Discovered a Simple Supplement That Causes Prostate Cancer Cells To Self-Destruct
Menadione, a vitamin K precursor, shows promise in slowing prostate cancer in mice by disrupting cancer cell survival processes, with potential applications for human treatment and myotubular myopathy...
scitechdaily.com
December 3, 2024 at 8:13 PM
Scientists Have Discovered a Simple Supplement That Causes Prostate Cancer Cells To Self-Destruct
Centronuclear Myopathies Drug Market – Global Industry Trends and Forecast to 2028 | Data Bridge Market Research www.databridgemarketresearch.com/reports/glob...
Centronuclear Myopathies Drug Market – Global Industry Trends and Forecast to 2028 | Data Bridge Market Research
Global Centronuclear Myopathies Drug Market, By Types (Myotubular Myopathy, Autosomal Centronuclear Myopathies and Others), Treatment (Night time Ventilation, Gastrostomy, Physiotherapy, Antibiotics a...
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January 9, 2025 at 6:20 PM
Centronuclear Myopathies Drug Market – Global Industry Trends and Forecast to 2028 | Data Bridge Market Research www.databridgemarketresearch.com/reports/glob...
The new issue of #ScienceTranslationalMedicine is out!
Clues from pediatricians' immune cells lead researchers to new antibody therapeutics for RSV and metapneumovirus, a newly approved anti-influenza drug shields ferrets from severe H5N1, and more. https://scim.ag/4qFzlYs
Clues from pediatricians' immune cells lead researchers to new antibody therapeutics for RSV and metapneumovirus, a newly approved anti-influenza drug shields ferrets from severe H5N1, and more. https://scim.ag/4qFzlYs
February 18, 2026 at 10:19 PM
The new issue of #ScienceTranslationalMedicine is out!
Clues from pediatricians' immune cells lead researchers to new antibody therapeutics for RSV and metapneumovirus, a newly approved anti-influenza drug shields ferrets from severe H5N1, and more. https://scim.ag/4qFzlYs
Clues from pediatricians' immune cells lead researchers to new antibody therapeutics for RSV and metapneumovirus, a newly approved anti-influenza drug shields ferrets from severe H5N1, and more. https://scim.ag/4qFzlYs
Our findings identify myosin dysfunction as a contributor to X-linked myotubular myopathy (XLMTM) – similarly to what we have seen in nemaline myopathy – and highlight myosin as a potential drug target.
@myofinlab.bsky.social
research.folkhalsan.fi/annual-repor...
@myofinlab.bsky.social
research.folkhalsan.fi/annual-repor...
May 20, 2026 at 6:31 AM
Our findings identify myosin dysfunction as a contributor to X-linked myotubular myopathy (XLMTM) – similarly to what we have seen in nemaline myopathy – and highlight myosin as a potential drug target.
@myofinlab.bsky.social
research.folkhalsan.fi/annual-repor...
@myofinlab.bsky.social
research.folkhalsan.fi/annual-repor...
Lack of myotubularin phosphatase activity is the main cause of X-linked Myotubular Myopathy
insight.jci.org/articles/vie...
insight.jci.org/articles/vie...
JCI Insight -
Lack of myotubularin phosphatase activity is the main cause of X-linked Myotubular Myopathy
insight.jci.org
October 15, 2025 at 4:27 PM
Lack of myotubularin phosphatase activity is the main cause of X-linked Myotubular Myopathy
insight.jci.org/articles/vie...
insight.jci.org/articles/vie...
Manejo multidisciplinar de la miopatía miotubular ligada al cromosoma X en España y Portugal: un análisis de una serie de casos www.sciencedirect.com/science/arti... #hvhebron #neuroped [Text complet]
Multidisciplinary management of X-linked myotubular myopathy in Spain and Portugal: A case series analysis
X-linked myotubular myopathy (XLMTM) is a severe, rare, familial neuromuscular disease caused by mutations in the MTM1 gene. XLMTM presents a wide spe…
www.sciencedirect.com
November 26, 2025 at 11:51 AM
Manejo multidisciplinar de la miopatía miotubular ligada al cromosoma X en España y Portugal: un análisis de una serie de casos www.sciencedirect.com/science/arti... #hvhebron #neuroped [Text complet]
New research shows that Menadione, a vitamin K precursor, slows prostate cancer in mice.
The researchers now hope to see the experiment translated to pilot studies in human prostate cancer patients.
scitechdaily.com/scientists-h...
The researchers now hope to see the experiment translated to pilot studies in human prostate cancer patients.
scitechdaily.com/scientists-h...
Scientists Have Discovered a Simple Supplement That Causes Prostate Cancer Cells To Self-Destruct
Menadione, a vitamin K precursor, shows promise in slowing prostate cancer in mice by disrupting cancer cell survival processes, with potential applications for human treatment and myotubular myopathy...
scitechdaily.com
November 30, 2024 at 5:14 PM
New research shows that Menadione, a vitamin K precursor, slows prostate cancer in mice.
The researchers now hope to see the experiment translated to pilot studies in human prostate cancer patients.
scitechdaily.com/scientists-h...
The researchers now hope to see the experiment translated to pilot studies in human prostate cancer patients.
scitechdaily.com/scientists-h...
There a number of stronger phenotypes associated with specific mutations. It would be good to connect with #MantonCenter, #MTM-CNMFamilyConnection, and Myotubular and Centronuclear Myopathy Patient Registry
February 10, 2025 at 1:20 PM
There a number of stronger phenotypes associated with specific mutations. It would be good to connect with #MantonCenter, #MTM-CNMFamilyConnection, and Myotubular and Centronuclear Myopathy Patient Registry
Here’s the paper I mean where I saw parallels pubmed.ncbi.nlm.nih.gov/17005396/
Extreme phenotypic variability in a German family with X-linked myotubular myopathy associated with E404K mutation in MTM1 - PubMed
X-linked myotubular myopathy (XLMTM) is a congenital muscle disorder caused by mutations in the MTM1 gene. Affected males usually present at birth with severe hypotonia and respiratory insufficiency, ...
pubmed.ncbi.nlm.nih.gov
February 9, 2025 at 11:03 PM
Here’s the paper I mean where I saw parallels pubmed.ncbi.nlm.nih.gov/17005396/