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FDA approves new treatment option for late-onset Pompe disease

LDRTC at WORLDSymposium™ 2023

LDRTC 's Poster Presentations:

Basic Science – Poster Session I – Wednesday, February 22, 3-4:00 PM
Poster# 184 Margarita Ivanova - Gender differences in circulating inflammatory, immune, & tissue growth markers associated with Fabry disease-related cardiomyopathy Kiosk# 22-A

Poster# 196 Neil Kasaci Caspase inhibitors can counteract inflammasome activation and caspase-1 mediated fibrosis in Fabry disease Kiosk# 24-A

Translational Research – Poster Session III – Thursday, February 23, 3-4:00 PM
Poster# 84 Julia Dao - TRAP5a and TRAP5b biomarkers in Gaucher disease Kiosk#8-A

Clinical Applications – Poster Session V – Friday, February 24, 3-4:00 PM
Poster# 138 Ozlem Goker-Alpan - The survival as relates to the clinical spectrum, molecular variants and chaperone response in acute neuronopathic Gaucher disease Kiosk#17-A

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FDA approves new treatment option for late-onset Pompe disease

LDRTC participates in studies that contributed to a new treatment option for late-onset Pompe disease

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LDRTC treated the first patient for the expanded access study

LDRTC treated the first patient for the expanded access study for Fabry disease

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Top 10% most cited paper published

Top 10% most cited paper published

Team LDRTC's article "Impaired autophagic and mitochondrial functions are partially restored by ERT in Gaucher and Fabry diseases" is among the top 10% most cited @PLOSONE papers published in 2019. #GaucherDisease #fabrydisease
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COVID-19 Vaccine Information for our Patients

Fairfax, VA - January 14, 2021

The Advisory Committee on Immunization Practices (ACIP) has issued interim recommendations for the use of Pfizer-BioNTech and Moderna COVID-19 vaccines for the prevention of coronavirus disease 2019 (COVID-19) in the United States. Please find the current information about these vaccines and some other relevant information for our patients. The below information is gathered from the CDC website and other peer-reviewed medical publications. 

Both COVID-19 vaccines are mRNA vaccines that target the spike glycoprotein of the SARS-CoV-2 virus. These vaccines are delivered using lipid nanoparticles (LNP), which prevent the degradation of mRNA. LNP or liposomes have been used to trap and protect the molecules until their target is reached (Vitiello at al, 2017). For vaccines, the target is an immune cell called an antigen-presenting cell. 

Pfizer BioNTech COVID-19 Vaccine: 16 years and older. 2-dose series separated by 21 days. A series started with the COVID-19 vaccine (Pfizer) should be completed with this product administered as an intramuscular (IM) injection in the (deltoid muscle) upper arm. 

Among vaccine recipients, reactogenicity symptoms, defined as local injection site or systemic reactions during the seven days after vaccination, were mostly mild to moderate. Systemic adverse reactions were more commonly reported after the second dose than after the first dose and were generally more frequent in persons aged 18–55. Systemic adverse reactions had an onset of 1–2 days after the vaccine and resolved in a day. Among vaccine recipients, the most side effects were fatigue, headache, muscle pain, chills, and injection site pain usually reported after the second dose. 

Moderna COVID-19 Vaccine: 18 years of age and older2-dose series separated by 28 days. A series started with COVID-19 vaccine (Moderna) should be completed with this product. Administered as an Intramuscular (IM) injection in the (deltoid muscle) upper arm.

Vaccination of persons with known current SARS-CoV-2 infection is preferentially deferred about 90 days after the initial infection. Thus, persons with documented acute SARS-CoV-2 infection within three months may delay vaccination until near the end of this period. 

Should our patients have a priority for COVID vaccines:

All our patients have chronic health problems, and some have serious medical issues. While each state decides about the vaccine’s distribution and administration, the current recommendation is over age 65, and those with underlying health risks should get priority over the general population. If you have serious health risks such as heart disease, kidney disease, obesity, chronic lung disease, or immune problems, you should get vaccinated as soon as you can.  

While these vaccines are found to be effective for prevention of COVID-19 infections, there is not yet enough long-term data for prevention of hospitalizations and other long term COVID-19 complications. We advise that our patients continue with the local and CDC guidelines for wearing masks, social distancing, and other hygiene precautions.