Menu

Patient Care Center

Menu

Author name: Nasir

Acoramidis Reduces All-Cause, Cardiovascular Mortality at 30 Months in ATTR-CM

Leave a Comment / News & Meetings / By

The 30-month results of the phase 3 ATTRibute-CM trial provide the latest insight into the effects of acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM) ahead of a potential regulatory submission for the agent. Presented during a hot line session at the European Society of Cardiology (ESC) Congress 2023, results of the trial suggest the acoramidis cohort experienced …

Acoramidis Reduces All-Cause, Cardiovascular Mortality at 30 Months in ATTR-CM Read More »

Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting

Leave a Comment / News & Meetings / By

– Cardiovascular and Renal Drugs Advisory Committee Voted 9:3 That the Benefits of Patisiran Outweigh its Risks for the Treatment of the Cardiomyopathy of ATTR Amyloidosis – – Prescription Drug User Fee Act Target Action Date is October 8, 2023 – CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep. 13, 2023– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics …

Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting Read More »

Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Leave a Comment / News & Meetings / By

– FDA Cites Insufficient Evidence of Clinical Meaningfulness – – No Clinical Safety, Study Conduct, Drug Quality or Manufacturing Issues Identified – – CRL Does Not Pertain to, nor Impact Commercial Availability of, ONPATTRO® (patisiran) for Existing Indication for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis in Adults – – Alnylam to Host …

Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis Read More »

Landmark CRISPR Trial Shows Promise Against Deadly Disease

Leave a Comment / Clinical Trial / By

Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease. The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver …

Landmark CRISPR Trial Shows Promise Against Deadly Disease Read More »

CRISPR Injected Into the Blood Treats a Genetic Disease for First Time

Leave a Comment / Clinical Trial / By

The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an enormous hurdle: getting the molecular scissors into the body and having it slice DNA in the tissues where it’s needed. Now, in a medical first, researchers have injected a CRISPR …

CRISPR Injected Into the Blood Treats a Genetic Disease for First Time Read More »

Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders

Leave a Comment / Clinical Trial / By

People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually builds up in the heart and nerves. Symptoms include numbness in the hands and feet, loss of control of the bowel and bladder and immobility. The condition gets progressively worse and is …

Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders Read More »

New Era of Medicine: Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans

Leave a Comment / Clinical Trial / By

Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and efficacy of in vivo CRISPR genome editing in humans. The team of researchers based in the U.S., the U.K., and New Zealand showed in a study that the companies’ lead in vivo genome editing candidate …

New Era of Medicine: Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans Read More »

UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

Leave a Comment / Clinical Trial / By

The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of DNA to be added, removed, or edited. NTLA-2001 is designed to eliminate the TTR gene from the DNA of liver cells, which would reduce TTR production significantly and prevent the …

UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001 Read More »

Informed Consent for Clinical Trials

Leave a Comment / Clinical Trial / By

The term informed consent is mistakenly viewed as the same as getting a research participant’s signature on the consent form. FDA believes that obtaining a research participant’s verbal or written informed consent is only part of the process. Informed Consent Involves Providing a Potential Participant With: Adequate information to allow for an informed decision about …

Informed Consent for Clinical Trials Read More »

Scroll to Top