W for Wilson Disease
Wilson disease is a rare, hereditary genetic ailment that impairs the body’s capacity to metabolize copper, resulting in its toxic buildup in essential organs such as the liver, brain, and eyes. Wilson disease results from mutations in the ATP7B gene, which facilitates the transport of excess copper from the liver to the bile for excretion […]
How Diet Coke causes atherosclerotic plaque – mechanism unlocked!!
It is known that high consumption of artificial sweeteners like aspartame can increase the risk of cardiovascular events but how it actually contributes to the disease is not clear. In the present study published in Cell Metabolism, the authors demonstrated its molecular mechanism in mice. They showed that an aspartame-rich diet in mice increased blood […]
U for Uveal Melanoma
Uveal melanoma, also known as ocular melanoma, is rare cancer that affects the uvea, the central layer of the eye. The uvea consists of the iris (the colored part of the eye), the ciliary body (which produces fluid for the eye), and the choroid (a layer of blood vessels under the retina). This disorder most […]
SYMBRAVO, A Fast-Acting, Dual-Action Drug to Relieve Migraine
Axsome Therapeutics’ SYMBRAVO was recently approved by the U.S. Food and Drug Administration (FDA). It combines a fast-acting serotonin receptor agonist, rizatriptan with meloxicam, a potent COX-2 preferential, nonsteroidal anti-inflammatory drug (NSAID). Using Molecular Solubility Enhanced Inclusion Complex (MoSEIC) rapid-absorption technology to speed up meloxicam absorption four times faster, SYMBRAVO offers quick relief for acute […]
T For Tay Sach’s Disease
Tay-Sachs disease (TSD) is an inherited rare autosomal recessive lysosomal disease caused by mutations in the Hexosaminidase A(Hex-A) gene, causing deficiency of Hex-A enzyme. This shortage leads an accumulation of GM2 gangliosides in the nervous system. TSD can be categorized into three types: Infantile, Juvenile, or Late Onset. Infantile and Juvenile forms cannot be treated […]
Semaglutide: A Breakthrough in Obesity Treatment
Introduction Obesity continues to challenge global health systems, necessitating innovative and effective therapies. Novo Nordisk’s latest development in the STEP UP clinical program highlights the potential of semaglutide 7.2 mg as a transformative solution for weight management. With significant weight loss results and a well-tolerated safety profile, this once-weekly GLP-1 receptor agonist demonstrates promise for […]
Based on Phase 3 trial data, the FDA approved Alhemo for the treatment of hemophilia-related bleeding
On December 20, 2024, FDA approved Alhemo (concizumab-mtci) injection to reduce the hemophilia related bleeding frequency in patients of 12 years and older. This is a first of its kind subcutaneous injection treatment for patients suffering from hemophilia A with factor 8 inhibitors or hemophilia B with factor 11 inhibitors Who is the treatment target? […]
Semaglutide Enhances Glycemic Control with Automated Insulin Delivery in Type 1 Diabetes
Introduction Managing type 1 diabetes (T1D) requires precise glucose control to minimize complications and improve quality of life. Automated insulin delivery (AID), which integrates continuous glucose monitoring (CGM) with algorithm-driven insulin dosing, is the current pinnacle of insulin therapy. However, achieving optimal glycemic control remains a challenge for many patients. A recent study published in […]
Scientists explained why you gain weight quickly after stopping dieting
I bet all of you have observed that you gain weight quickly after a weight loss when you stop dieting or exercising. A group of scientists from Switzerland have recently found molecular evidence of this phenomenon. In a recent research article published in Nature, they found similar epigenetic signatures on fat cells (adipocytes) during obesity […]
Avoiding sugars in the first 1000 days of life can reduce the risk of type 2 diabetes and hypertension in your 60s – A World War II sugar rationing study
A recent study published in the Science journal showed that limiting sugar up to 1000 days from conception can reduce the risk and delay the onset of type 2 diabetes (T2D) and hypertension later in life, in their 60s. Study cohort This study was based on a UK cohort of 60,183 participants born between October […]
S for Sickle Cell Disease: Part II
Sickle Cell Disease (SCD) is a genetic disorder that causes the production of abnormally shaped red blood cells, which are rigid, sickle-shaped, and can block blood flow. This leads to severe pain (sickle cell crises), organ damage (including liver, heart, and spleen), and a reduced life expectancy. Treatments like blood transfusions, pain management, and bone […]
FDA Approves Obecabtagene Autoleucel for Advanced B-Cell Acute Lymphoblastic Leukemia
Introduction The FDA’s recent approval of obecabtagene autoleucel (Aucatzyl) marks a significant milestone in immunotherapy, offering hope for adults battling relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Developed by Autolus Inc., this CD19-directed, genetically modified autologous T-cell therapy targets a critical unmet need in hematologic oncology. Current Regulatory Status Obecabtagene autoleucel received FDA […]
R For Retinoblastoma- Part I
Retinoblastoma is a rare eye cancer that primarily affects young children, typically diagnosed before the age of five. It originates in the retina, the light-sensitive tissue at the back of the eye, where nerve cells undergo changes leading to tumor formation. While most cases occur in children, it can also appear in adults. Researchers estimate […]
FDA Certifies Vyloy as a Novel Approach Against Gastric Cancer: a CLDN18.2-Targeted Therapy
On October 18, 2024, FDA approved Vyloy (zolbetuximab-clzb) as the first and only claudin-18.2-targeted antibody to treat gastric cancer. The drug has been approved to be used in combination with fluoropyrimidine and platinum containing chemotherapy for patients suffering from locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction […]
FDA Approves Zolbetuximab-clzb for Advanced Gastric and GEJ Adenocarcinoma
IntroductionThe U.S. Food and Drug Administration (FDA) has recently approved a breakthrough treatment, zolbetuximab-clzb (Vyloy, Astellas Pharma US, Inc.), for the first-line treatment of adults with locally advanced, unresectable, or metastatic gastric and gastroesophageal junction (GEJ) adenocarcinoma. Targeting a specific biomarker—claudin 18.2 (CLDN18.2)—this therapy, combined with chemotherapy, offers a promising new option for patients with […]
FDA Approval of Inavolisib Combination Therapy for PIK3CA-Mutated, HR-Positive, HER2-Negative Breast Cancer
IntroductionThe FDA has recently approved a new combination therapy, marking a significant advance in the treatment of endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, advanced breast cancer. This combination involves inavolisib (Itovebi), palbociclib, and fulvestrant, offering new hope to patients with recurrent disease after adjuvant endocrine therapy. Current Regulatory StatusInavolisib, […]
Blood glucose as a predictor of cardiovascular events
A European observational study has determined that 2h post-load glucose (2-hPG) is a better marker than HbA1c in predicting cardiovascular events for coronary artery disease patients. This study was conducted in various European countries and was published in the Lancet Diabetes Endocrinology in Sept 2024. Coronary artery disease is a condition where fat, cholesterol and other materials […]
VYALEV approved by FDA for Advanced stage Parkinson’s Disease patients
AbbVie’s subcutaneous 24-hour continuous infusion drug, VYALEV is now approved by the U.S. Food and Drug Administration (FDA) to reduce motor fluctuations in advanced stage Parkinson’s Disease patients. This is the first and only subcutaneous continuous infusion therapy that involves wearing a portable kit throughout the day allowing personalized dosing based on patient’s need. Also […]
Q for Q Fever
Q fever is a rare infectious disease transmitted through the inhalation or ingestion of the bacteria Coxiella burnetii. Cattle, sheep, and goats frequently serve as bacterial hosts for this microorganism. C. burnetii is mostly transmitted through inhalation of contaminated air or ingestion of contaminated food and beverages. Agricultural laborers, particularly those engaged with livestock, individuals […]
P for Progeria: Part II
Progeria or Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare genetic disorder characterized by accelerated aging. Caused by a mutation in the LMNA gene, it leads to the accumulation of the toxic protein Progerin, resulting in severe cardiovascular issues and a life expectancy averaging 14.5 years. Progerin disrupts nuclear structure and function, causing various cellular abnormalities. […]
P for Progeria- Part I
Progeria or Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare and fatal genetic disorder characterized by premature aging that begins in childhood. The condition is typically not inherited but arises from a sporadic autosomal dominant mutation, with a low chance of occurrence (1 in 4 to 8 million), increasing to 2-3% if a family has already […]
Nivolumab Approved for Neoadjuvant and Adjuvant Therapy in Resectable NSCLC: A New Horizon for Lung Cancer Treatment
Introduction In a significant milestone for lung cancer treatment, the FDA has approved nivolumab, in combination with platinum-doublet chemotherapy, as a neoadjuvant therapy for resectable non-small cell lung cancer (NSCLC). Following surgery, nivolumab is administered as a single-agent adjuvant treatment. This approval is specifically for adults with tumors ≥4 cm and/or node-positive NSCLC without EGFR […]
First iPSC- Islet Transplantation in Humans- Proceed with Caution
For the first time, a group of scientists from China successfully performed islet transplantation using iPSCs (induced pluripotent stem cells) in one patient. They made iPSCs from the fat cells of the patients using chemicals instead of the conventional way of using Yamanaka factors. They successfully tested these human iPSCs-derived islets on monkeys before using […]
O for Osteosarcoma- Part II
The future of osteosarcoma therapy is centered on innovative clinical trial designs that prioritize progression-free survival and the identification of potentially effective agents, such as regorafenib and cabozantinib. These trials aim to explore targeted combinations in patients with tumors that have been genomically and immunologically characterized. Recent advances in genetic sequencing have uncovered significant complexity […]
Wegovy®: Expanding Benefits for Obesity-Related Heart Failure with Preserved Ejection Fraction
Introduction Obesity-related heart failure with preserved ejection fraction (HFpEF) presents a significant health challenge, particularly in those with obesity and type 2 diabetes. Wegovy® (semaglutide 2.4 mg), a once-weekly subcutaneous injection, is now at the forefront of an important advancement. Recently, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a […]
Recent FDA Approvals pack Double punches against Niemann-Pick disease type C
Rare diseases, affecting fewer than 200,000 people in the U.S., pose significant challenges due to their low prevalence. According to the NIH, there are over 7,000 known rare diseases, each with unique symptoms and treatments. For example, cystic fibrosis, a genetic disease affecting the lungs, often goes misdiagnosed. The lack of large patient populations hinders […]
Transparent mice are here – Not a science fiction, but a reality.
Scientists at Stanford University have made live mice transparent, easing biomedical research and saving their lives. They used an FDA-approved food coloring dye called tartrazine, which makes the mice’s skin transparent. They used this dye on the mouse brain, abdomen, and muscle for imaging. Below is the figure of the mouse’s abdomen before and after […]
Nanotechnology: Revolutionizing Diabetes Treatment and Diagnostics
The increasing global prevalence of diabetes demands innovative solutions beyond traditional treatments, which often struggle with high costs and limited efficacy. Nanotechnology, a rapidly evolving field, offers groundbreaking possibilities in managing diabetes, providing more targeted, effective, and cost-efficient interventions. Image Source: Vinoth Khandelwal Nanotechnology in Diabetes Management Nanomedicine involves the use of nanoparticles (NPs), whose […]
O for Osteosarcoma – Part I
Osteosarcoma is a rare malignancy. Annually, approximately 1,000 new osteosarcoma cases are identified in the United States. Approximately fifty percent of these cases involve children and adolescents. Most osteosarcomas manifest in children, adolescents, and young adults aged 10 to 30. Adolescents are the most frequently impacted demographic, although individuals of any age may acquire osteosarcoma. […]
Once-Weekly Insulins Show Promise in Simplifying Diabetes Management
Introduction Managing type 2 diabetes with insulin therapy presents challenges, particularly with daily injections. Recently, new clinical studies have emerged evaluating once-weekly insulin options that aim to reduce the burden of daily injections while maintaining effective blood sugar control. These innovative treatments, including once-weekly icodec (Awiqli, Novo Nordisk) and insulin efsitora alfa (efsitora, Eli Lilly), […]
