AZLatina
@endrarediseases.bsky.social
Blue Dog Dem. Rare Disease Patient Advocate for Amyloidosis that killed my Mom & ALS that is killing my friends. Passionate about Enviro Justice, Foster Care & Gun Safety. Proud GGD of a Righteous Gentile murdered by Nazis. 💙LA sports.
Pinned
AZLatina
@endrarediseases.bsky.social
· Nov 17
People with terminal #RareDiseases urgently need DISRUPTIVE policy change & a conditional approval pathway at FDA.
32k people with 100% fatal #ALS like Brian Wallach are #DyingWaiting when drugs could help them live.
Humanity & science can co-exist.
#EndALS
@bsw5020.bsky.social
32k people with 100% fatal #ALS like Brian Wallach are #DyingWaiting when drugs could help them live.
Humanity & science can co-exist.
#EndALS
@bsw5020.bsky.social
Reposted by AZLatina
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy; muscle performance tests in small trial seen as step to accelerated approval
www.statnews.com/2024/11/18/d...
www.statnews.com/2024/11/18/d...
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy for Duchenne muscular dystrophy.
www.statnews.com
November 18, 2024 at 12:42 PM
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy; muscle performance tests in small trial seen as step to accelerated approval
www.statnews.com/2024/11/18/d...
www.statnews.com/2024/11/18/d...
Reposted by AZLatina
Encouraging sign as Capricor seeks FDA approval of deramiocel for Duchenne Muscular Dystrophy based on encouraging data ipscell.com/2024/11/capr... #stemcells
Capricor seeks FDA approval of deramiocel for Duchenne based on encouraging data - The Niche
Stem cell biologist discusses very encouraging data from Capricor on Duchenne Muscular Dystrophy and future prospects.
ipscell.com
November 8, 2024 at 4:21 PM
Encouraging sign as Capricor seeks FDA approval of deramiocel for Duchenne Muscular Dystrophy based on encouraging data ipscell.com/2024/11/capr... #stemcells
Reposted by AZLatina
New #BrainDev journal paper: Safety and efficacy of viltolarsen treatment in patients with Duchenne muscular dystrophy: A retrospective study with 3-year follow-up. #DMD. tinyurl.com/3eurb55v
Safety and efficacy of viltolarsen treatment in patients with Duchenne muscular dystrophy: A retrospective study with 3-year follow-up
Duchenne muscular dystrophy (DMD) is a hereditary neuromuscular disorder characterized by severe, progressive muscle wasting. Viltolarsen, a formulati…
tinyurl.com
November 17, 2024 at 4:58 PM
New #BrainDev journal paper: Safety and efficacy of viltolarsen treatment in patients with Duchenne muscular dystrophy: A retrospective study with 3-year follow-up. #DMD. tinyurl.com/3eurb55v
Reposted by AZLatina
Mayo clinic liver Amyloid series
62% AL
25% ALECT2
7% AApo AI
4% AA
2% ATTR
1% lysozyme
62% AL
25% ALECT2
7% AApo AI
4% AA
2% ATTR
1% lysozyme
November 16, 2024 at 10:59 PM
Mayo clinic liver Amyloid series
62% AL
25% ALECT2
7% AApo AI
4% AA
2% ATTR
1% lysozyme
62% AL
25% ALECT2
7% AApo AI
4% AA
2% ATTR
1% lysozyme
Reposted by AZLatina
Structure-based probe reveals the presence of large transthyretin aggregates in plasma of ATTR amyloidosis patients https://www.biorxiv.org/content/10.1101/2024.03.09.584228v1
Structure-based probe reveals the presence of large transthyretin aggregates in plasma of ATTR amyloidosis patients https://www.biorxiv.org/content/10.1101/2024.03.09.584228v1
ATTR amyloidosis is a relentlessly progressive disease caused by the misfolding and systemic accumul
www.biorxiv.org
March 11, 2024 at 7:20 AM
Structure-based probe reveals the presence of large transthyretin aggregates in plasma of ATTR amyloidosis patients https://www.biorxiv.org/content/10.1101/2024.03.09.584228v1
Reposted by AZLatina
Scientists uncover cerebrospinal fluid markers for Alzheimer's detection and treatment 🧬🧠🔬 www.news-medical.net/news/2024111... #Alzheimers #Genomics #Proteomics #BrainHealth #CSFresearch #Neuroscience #GeneticMapping #DrugDiscovery #Biomarkers #Neurogenomics
Scientists uncover cerebrospinal fluid markers for Alzheimer's detection and treatment
Researchers map the unique genetic regulation of proteins in human cerebrospinal fluid, identifying potential targets for Alzheimer’s treatments.
www.news-medical.net
November 13, 2024 at 3:16 AM
Scientists uncover cerebrospinal fluid markers for Alzheimer's detection and treatment 🧬🧠🔬 www.news-medical.net/news/2024111... #Alzheimers #Genomics #Proteomics #BrainHealth #CSFresearch #Neuroscience #GeneticMapping #DrugDiscovery #Biomarkers #Neurogenomics
Reposted by AZLatina
Reposted by AZLatina
The MND Association, My Name'5 Doddie Foundation and Darby Rimmer Foundation have funded our project with the Mario Negri Institute in Italy to study whether there is a link between #ALS #MND and professional football. Read about it here:
www.mndassociation.org/media/latest...
@ukmndri.bsky.social
www.mndassociation.org/media/latest...
@ukmndri.bsky.social
New research project investigates potential link between professional football and MND
New research project investigates potential link between professional football and MND
www.mndassociation.org
November 16, 2024 at 11:40 AM
The MND Association, My Name'5 Doddie Foundation and Darby Rimmer Foundation have funded our project with the Mario Negri Institute in Italy to study whether there is a link between #ALS #MND and professional football. Read about it here:
www.mndassociation.org/media/latest...
@ukmndri.bsky.social
www.mndassociation.org/media/latest...
@ukmndri.bsky.social
People with terminal #RareDiseases urgently need DISRUPTIVE policy change & a conditional approval pathway at FDA.
32k people with 100% fatal #ALS like Brian Wallach are #DyingWaiting when drugs could help them live.
Humanity & science can co-exist.
#EndALS
@bsw5020.bsky.social
32k people with 100% fatal #ALS like Brian Wallach are #DyingWaiting when drugs could help them live.
Humanity & science can co-exist.
#EndALS
@bsw5020.bsky.social
November 17, 2024 at 9:55 PM
People with terminal #RareDiseases urgently need DISRUPTIVE policy change & a conditional approval pathway at FDA.
32k people with 100% fatal #ALS like Brian Wallach are #DyingWaiting when drugs could help them live.
Humanity & science can co-exist.
#EndALS
@bsw5020.bsky.social
32k people with 100% fatal #ALS like Brian Wallach are #DyingWaiting when drugs could help them live.
Humanity & science can co-exist.
#EndALS
@bsw5020.bsky.social