L for Legg-Calve-Perthes Disease- Part II
Legg-Calvé-Perthes disease (LCPD) is a pediatric hip condition characterized by interruption of the blood supply to the femoral head, leading to osteonecrosis. This condition primarily affects children between 4 and 8 years but can occur between 2 and 15 years. Various treatments, including both surgical and nonsurgical options, have been explored to manage LCPD. Despite […]
FDA Approves Neoadjuvant/Adjuvant Durvalumab for Resectable NSCLC
Introduction The U.S. Food and Drug Administration (FDA) recently approved the use of durvalumab (Imfinzi, AstraZeneca) in combination with platinum-based chemotherapy as a neoadjuvant treatment for adults with resectable non-small cell lung cancer (NSCLC). This treatment regimen is followed by durvalumab monotherapy as adjuvant therapy post-surgery. This approval is specifically for patients with tumors measuring […]
Tasty Traps: FDA and FTC Warn Against Copycat Snack Containing Delta-8 THC
FDA and FTC crack down on illegal delta-8 THC copycat snacks, protecting consumers from unregulated edibles in deceptive packaging that risks accidental ingestion, especially by children Imagine opening a yellow and orange pack of Flamin’ hot Cheetos and munching on one of those crunchy sticks. Instead of the cheesy, salty taste of nostalgia, you start […]
FDA Grants Accelerated Approval to Afamitresgene Autoleucel for Unresectable or Metastatic Synovial Sarcoma
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to afamitresgene autoleucel (TECELRA, Adaptimmune, LLC), a genetically modified autologous T cell immunotherapy. This approval targets adult patients with unresectable or metastatic synovial sarcoma, particularly those who have undergone prior chemotherapy and possess specific HLA-A alleles, with tumors expressing the melanoma-associated antigen A4 (MAGE-A4). […]
L for Legg- Calve-Perthes Disease- Part I
Legg-Calvé-Perthes Disease (LCPD) is a type of osteochondrosis affecting children, primarily between the ages of 2 and 12, with a higher incidence in those aged 5 to 6 and a greater prevalence in boys. This condition involves the degeneration and subsequent regeneration of the epiphysis, or growth end, of a bone. Specifically, LCPD impacts the […]
Let’s MASHup!!
MASH (Metabolic dysfunction associated steatohepatitis) is a severe form of non-alcoholic liver disease, previously known as NASH (Non-alcoholic steatohepatitis). It is caused by increased fat deposits and inflammation in the liver despite low alcohol consumption. This further leads to liver scarring called cirrhosis. Untreated cirrhosis causes irreversible damage to the liver leading to its failure […]
K for Kennedy Disease- Part II
Kennedy’s disease, also known as Spinal and Bulbar Muscular Atrophy (SBMA), first identified over two decades ago due to a mutation that expands a CAG trinucleotide repeat in the androgen receptor gene, results in an androgen receptor protein with an extended polyglutamine sequence. These disorders are characterized by protein aggregation, mitochondrial dysfunction, and transcriptional abnormalities. […]
FDA Expands Endometrial Cancer Indication for Dostarlimab-gxly with Chemotherapy
The Food and Drug Administration (FDA) has approved a new treatment regimen for adult patients with primary advanced or recurrent endometrial cancer (EC). This approval includes the combination of dostarlimab-gxly (Jemperli, GSK) with carboplatin and paclitaxel, followed by single-agent dostarlimab-gxly. This regimen was previously approved for patients with mismatch repair deficient (dMMR) or microsatellite instability-high […]
A Ray of Hope- Islets transplantation for T2D patients too
Islets are a group of hormone producing cells that control blood glucose levels. Islet transplantation is considered a cure for type 1 diabetes (T1D). T1D is caused by the destruction of beta cells in islets by the body’s immune system. These beta cells produce insulin that decreases blood glucose. Islet transplantation is challenging due to […]
FDA Approves Daratumumab and Hyaluronidase-fihj Combination for Multiple Myeloma
The Food and Drug Administration (FDA) has approved the combination of daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen) with bortezomib, lenalidomide, and dexamethasone for the induction and consolidation treatment of newly diagnosed multiple myeloma patients eligible for autologous stem cell transplant (ASCT). Image Source: Vinoth Khandelwal This approval was based on the results of the PERSEUS […]
K For Kennedy Disease- Part I
Kennedy Disease, also known as Spinal Bulbar Muscular Atrophy (SBMA), is a rare X-linked recessive genetic progressive neuromuscular disease that causes muscle weakness and wasting (atrophy) throughout the body. Symptoms typically begin to appear between the ages of 30 and 50, with life expectancy being nearly normal. Males with the inherited gene usually develop symptoms, […]
J for Juvenile CLN3 Disease- Part II
Juvenile CLN3 disease, also known as Batten disease, currently has no cure, so specialized symptom management and therapy are crucial for maintaining the quality of life for affected children, young people, and their families. Comprehensive support for parents, siblings, and extended family members is essential. Therapeutic approaches to Batten disease ranges from – Enzyme Replacement […]
Slimmer Days Ahead? Roche’s New Obesity Drug Shows Potential
Roche’s oral GLP-1 receptor agonist demonstrates significant weight loss in Phase I study. The global prevalence of obesity has surged dramatically over the past half century, driving a corresponding increase in pharmaceutical development within this therapeutic area. This week, Roche introduced a new treatment option, marking a significant advancement in the management of obesity. Facts […]
Merck Signs MoU with Gene Therapy Research Institution to Advance Gene Therapy Production
Merck, a prominent science and technology company, has recently signed a non-binding Memorandum of Understanding (MoU) with Gene Therapy Research Institution Co., Ltd. (GTRI), a Japanese clinical-stage biotech start-up focused on developing gene therapies using adeno-associated virus (AAV) vectors. This collaboration marks one of the first clinical-stage applications of Merck’s Sf-RVN® Insect Cell Line for […]
Alkaline Phosphatase: Potential cure for acute kidney injury?
Acute kidney injury (AKI) affects 20-50% of all hospitalized patients. It is characterized by an increase in serum creatinine and decreased urine output. AKI has mortality rate of >50%. Many factors are responsible for AKI like trauma, cardiovascular surgery, administration of nephrotoxic drugs, but sepsis associated AKI is the prominent one. Current treatment plans include […]
Mito!! Give me the power to rejuvenate!
We all know that mitochondria are the powerhouse of cells because they generate energy from food. However, we did not know their involvement in cellular regeneration. Scientists at UT Southwestern Medical Center determined that liver cells with damaged mitochondria have less ability to regenerate when injured. Henceforth, allowing cells with undamaged mitochondria to grow more, […]
J For Juvenile CLN3 Disease
Juvenile CLN3 disease, commonly known as Juvenile Batten Disease, is an extremely rare and fatal inherited disorder primarily affecting the nervous system in children. The disease is caused by mutations in the CLN3 gene, which encodes the CLN3 protein. More than sixty-seven different mutations in the CLN3 gene have been identified as responsible for Juvenile […]
Forget Me Not: New Alzheimer’s Drug from Eli Lilly Gets FDA Nod
The FDA approved donanemab, a new drug for early-stage Alzheimer’s disease. I remember my grandmother, her summer house filled with the smell of freshly baked cookies, the air vibrating with the laughter of my cousins rolling on the floor from her silly jokes. It was the best time of our childhood. I wish it remained […]
FDA Grants Accelerated Approval to Epcoritamab-bysp for Relapsed or Refractory Follicular Lymphoma
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to epcoritamab-bysp (Epkinly), developed by Genmab, for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This approval marks a significant advancement in the treatment options for FL, a type of non-Hodgkin lymphoma. […]
I for I-Cell Disease- Part II
I-cell disease or Mucolipidosis Type II (MLII) is a devastating rare genetic disorder characterized by severe multisystemic involvement and a limited lifespan. It is a severe lysosomal storage disorder caused by mutations in the GNPTAB gene located on chromosome 12q23.2. This gene encodes the alpha and beta subunits of N-acetylglucosamine-1-phosphotransferase (GlcNAc-1-phosphotransferase), which plays a crucial […]
FDA Grants Accelerated Approval to Adagrasib with Cetuximab for KRAS G12C-Mutated Colorectal Cancer
The Food and Drug Administration (FDA) has granted accelerated approval for the combination of adagrasib (Krazati; Mirati Therapeutics, Inc.) and cetuximab for the treatment of adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC). This approval applies to patients who have previously been treated with chemotherapy regimens based on fluoropyrimidine, oxaliplatin, and irinotecan, […]
I for I-Cell Disease- Part I
I-cell disease, also known as Mucolipidosis type II, is a severe rare lysosomal storage disorder caused by mutations in the GNPTAB gene located on chromosome 12q23.2. This gene encodes the alpha and beta subunits of N-acetylglucosamine-1-phosphotransferase, which plays a critical role in processing and packaging lysosomal enzymes by tagging them with mannose-6-phosphate in the Golgi […]
Iqirvo Clears FDA Hurdle, Offering New Relief for Primary Biliary Cholangitis Patients
The FDA approves Iqirvo (elafibranor) for primary biliary cholangitis (PBC) treatment, offering a new option for patients intolerant to UDCA. What is Primary Biliary Cholangitis (PBC)? A case study from December 2023 reports a 19-year-old male with multiple congenital abnormalities and an intellectual disability suffered from pruritus for 5 years. Tests revealed elevated levels of […]
H for Huntington’s Disease- Part II
Huntington’s disease (HD) is a debilitating neurodegenerative condition caused by an abnormal expansion of CAG triplets in the huntingtin (HTT) gene, leading to the progressive deterioration of GABAergic neurons in the brain’s central regions. As of now, there is no cure for HD, and treatment focuses on symptom management and improving quality of life. The […]
FDA Approves Blinatumomab as Consolidation Therapy for CD19-Positive Philadelphia Chromosome-Negative B-Cell Precursor Acute Lymphoblastic Leukemia
The Food and Drug Administration (FDA) has granted approval for blinatumomab (Blincyto, Amgen) for use in adult and pediatric patients aged one month and older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (Ph-negative BCP ALL) during the consolidation phase of multiphase chemotherapy. This decision marks a significant advancement in the treatment regimen for […]
H for Huntington’s Disease – Part I
Huntington’s disease (HD) is a rare, inherited neurodegenerative disorder affecting the central nervous system. Characterized by involuntary movements, behavioral disturbances, and dementia, HD typically manifests between ages 30 and 50, though juvenile cases also occur. Symptoms include chorea, dystonia, cognitive decline, psychiatric issues, and difficulties with speech and swallowing. The disease leads to complete dependency […]
FDA Grants Accelerated Approval to Repotrectinib for NTRK Fusion-Positive Tumors
The Food and Drug Administration (FDA) has granted accelerated approval to repotrectinib (AUGTYRO, Bristol-Myers Squibb; BMS) for the treatment of adult and pediatric patients aged 12 and older with solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. This approval covers patients with locally advanced or metastatic tumors for whom surgical resection would […]
Breaking Barriers in Rare Disease Treatment: FDA Approves Bkemv as First Interchangeable Biosimilar
The US Food and Drug Administration (FDA) has approved Amgen’s Bkemv as the first interchangeable biosimilar to Soliris for treating rare diseases, such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The FDA has approved Bkemv, a biosimilar, as the first interchangeable treatment for two rare and debilitating diseases: paroxysmal nocturnal hemoglobinuria […]
US FDA Approves Breyanzi for Relapsed or Refractory Mantle Cell Lymphoma
The U.S. Food and Drug Administration (FDA) has approved lisocabtagene maraleucel (Breyanzi), developed by Juno Therapeutics, Inc., for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have undergone at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor (BTKi). This approval marks a significant advancement […]
G for Gaucher Disease – Part II
Gaucher disease results from mutations in the GBA gene, disrupting the breakdown of glucocerebroside and causing its accumulation, leading to cellular damage. Efforts to repair this faulty “switch” have long been pursued in medical research. This disease though currently incurable, can be managed effectively with available therapies. Type 1 Gaucher patients can lead full lives, […]
