It Was All a Dream
Editor's Note: Patient Worthy is honored to share Michelle Patidar's patient story, provided to us by our friends at Elephants & Tea. To see the article in its original format,…
Continue Reading
It Was All a Dream
From Cancer Treatment to Childhood Health: How Foresee’s Injectable Drug Addresses a Rare Pediatric Challenge
When pharmaceutical companies develop medications for serious diseases like prostate cancer, few anticipate how those treatments might transform care for entirely different patient populations. Yet, according to FiercePharma.com, Foresee Pharmaceuticals…
Continue Reading
From Cancer Treatment to Childhood Health: How Foresee’s Injectable Drug Addresses a Rare Pediatric Challenge
CRISPR Therapeutics Shares Promising Updates on Zugo-cel in Autoimmune and Oncology Trials
CRISPR Therapeutics has announced encouraging early results for zugocaptagene geleucel (zugo-cel), its investigational allogeneic CAR T therapy targeting CD19, in both autoimmune diseases and hematologic malignancies. Autoimmune Disease Progress In…
Continue Reading
CRISPR Therapeutics Shares Promising Updates on Zugo-cel in Autoimmune and Oncology Trials
FDA Approves Amneal’s Denosumab Biosimilars Boncresa and Oziltus
As reported on PharmaBiz, Amneal Pharmaceuticals, in collaboration with mAbxience, has received U.S. Food and Drug Administration (FDA) approval for two denosumab biosimilars: Boncresa (referencing Prolia) and Oziltus (referencing Xgeva).…
Continue Reading
FDA Approves Amneal’s Denosumab Biosimilars Boncresa and Oziltus
FDA Grants Full Approval to Rucaparib for BRCA-Mutated mCRPC
In a recent statement, the U.S. Food and Drug Administration (FDA) has authorized regular approval to rucaparib (Rubraca) for adults with metastatic castration-resistant prostate cancer (mCRPC) harboring deleterious BRCA mutations—either…
Continue Reading
FDA Grants Full Approval to Rucaparib for BRCA-Mutated mCRPC
What’s Missing From Many Surgeries? The Answer Might Be Your Nerves
There's a lot to consider when you’re facing tumor removal surgery. Think about mandible or jaw surgery, for example. Your face is part of every interaction you have, how you…
Continue Reading
What’s Missing From Many Surgeries? The Answer Might Be Your Nerves
Is Your Mental Diet Helping Or Hurting Your Health?
When we hear the word diet, most of us automatically think of food. But what about our thoughts? Let’s look at them in terms of consumption as well. According to…
Continue Reading
Is Your Mental Diet Helping Or Hurting Your Health?
FDA Panel Sparks Debate Over Antidepressant Use in Pregnancy
As featured on KFF News, Heidi DiLorenzo was preparing to welcome her second child, and her biggest concern wasn’t taking Zoloft. The Birmingham attorney had relied on the medication for…
Continue Reading
FDA Panel Sparks Debate Over Antidepressant Use in Pregnancy
Hereditary Fructose Intolerance (HFI) UK – an HFI Support Charity
This charity has been set up to offer research based and lived experience, advice and support. Aims: To support any individual of any age with HFI, particularly parents of babies…
Continue Reading
Hereditary Fructose Intolerance (HFI) UK – an HFI Support Charity
Her Life Changed at a Red Light. Life and Love with HIV is Possible.
My name is Jessica Glaspie, and I have been living with HIV for over 15 years! A lot of people think that life and love with HIV is almost impossible…
Continue Reading
Her Life Changed at a Red Light. Life and Love with HIV is Possible.
An Unexpected Liver Transplant Part 4 – The Journey to Disney World
“We’re going to Disney World!” That's what all the winners say, right? When the big game is over and the team has won the championship, the winners say, for a…
Continue Reading
An Unexpected Liver Transplant Part 4 – The Journey to Disney World
FDA Unveils TEMPO Pilot to Advance Digital Health for Chronic Disease Care
In a recent statement, The U.S. Food and Drug Administration (FDA) has introduced the Technology-Enabled Meaningful Patient Outcomes (TEMPO) Pilot, a groundbreaking initiative aimed at expanding access to digital health…
Continue Reading
FDA Unveils TEMPO Pilot to Advance Digital Health for Chronic Disease Care
Human Gut Cells Reveal Unexpected Role in Celiac Disease
As reported by MedicalXpress, Researchers have uncovered a surprising immune function in specialized intestinal cells that may reshape our understanding of gut health and autoimmune disorders. The study, published in…
Continue Reading
Human Gut Cells Reveal Unexpected Role in Celiac Disease
NYU Langone Doctors Transplanted a Pig Kidney into a Brain Dead Patient Reversing Rejection Twice
A team of doctors at NYU Langone transplanted a pig kidney into a brain-dead patient and reversed its rejection twice during the 61-day study. Currently, estimates of the number of…
Continue Reading
NYU Langone Doctors Transplanted a Pig Kidney into a Brain Dead Patient Reversing Rejection Twice
The Ever growing Field of Molecular Glues
Breaking a system into small independent and interchangeable modules that can perform certain functions is called modularity. For many years the KRAS oncogene proved to be a classic example of…
Continue Reading
The Ever growing Field of Molecular Glues
Chiesi Group Partners with Aliada Therapeutics to Advance Blood-Brain Barrier Technology for Rare Diseases
Chiesi Group has entered an exclusive licensing agreement with Aliada Therapeutics, a subsidiary of AbbVie, to develop therapies that can cross the blood-brain barrier (BBB) for lysosomal storage disorders (LSDs).…
Continue Reading
Chiesi Group Partners with Aliada Therapeutics to Advance Blood-Brain Barrier Technology for Rare Diseases
PopEVE Aims to Fast-Track Rare Disease Diagnoses by Ranking Genetic Variant Severity
A new artificial intelligence model, popEVE, could accelerate diagnoses for rare, single-variant genetic diseases by scoring how likely each genetic change in a patient’s genome is to cause disease. Developed…
Continue Reading
PopEVE Aims to Fast-Track Rare Disease Diagnoses by Ranking Genetic Variant Severity
Houston Methodist Researchers Identify T Cells That Attack Transplanted Organs
The findings were recently published in Nature Immunology and reported by Precision Medicine suggesting that a factor called Interferon Regulatory Factor 4 (IRF4) takes responsibility as a driver of transplant…
Continue Reading
Houston Methodist Researchers Identify T Cells That Attack Transplanted Organs
CRISPR-Cas Technologies and the Potential for Revolutionizing Genetic Medicine
A gene-editing protein, small enough to fit into adeno-associated viruses (AAVs) that infect humans has been identified. As reported by Precision Medicine, these compact AAVs can be modified so that…
Continue Reading
CRISPR-Cas Technologies and the Potential for Revolutionizing Genetic Medicine
NORD Opens Applications for New Rare Disease Patient Registries
As reported on PharmaBiz, the National Organization for Rare Disorders (NORD) has announced a new request for proposals (RFP) aimed at nonprofit patient advocacy groups interested in launching rare disease…
Continue Reading
NORD Opens Applications for New Rare Disease Patient Registries
Comparing Expert Clinicians and AI in Diagnosing Rare Diseases
Why Rare Diseases Pose a Diagnostic Challenge As discussed in the Orphanet Journal of Rare Disease, a "rare disease" is defined as a condition affecting fewer than 1 in 2,000…
Continue Reading
Comparing Expert Clinicians and AI in Diagnosing Rare Diseases
Jim Wilson’s Pioneering Journey: Transforming Rare Disease Treatment Through Innovative Gene Therapy Part 2
Please go HERE to read Part 1 of this article The company, GEMMABIO, will pursue a number of rare, large indications. A new spinout, RareTx, will be dedicated to Orphan…
Continue Reading
Jim Wilson’s Pioneering Journey: Transforming Rare Disease Treatment Through Innovative Gene Therapy Part 2
Breakthrough FDA Approval Offers First-Ever Treatment for Life-Threatening Mitochondrial Energy Disorder
The U.S. Food and Drug Administration has approved UCB's Kygevvi, marking a historic milestone for patients with thymidine kinase 2 deficiency (TK2d), an ultra-rare inherited mitochondrial disorder that has lacked…
Continue Reading
Breakthrough FDA Approval Offers First-Ever Treatment for Life-Threatening Mitochondrial Energy Disorder
Researchers at University of Prague Unveil Lab-on-a-Scalpel
Professor Zdeněk Sofer of the University of Prague and his team developed and validated a "Lab-on-a-Scalpel" concept which is a surgical tool incorporating an integrated diagnostic sensor. Their findings were…
Continue Reading
Researchers at University of Prague Unveil Lab-on-a-Scalpel
Jim Wilson’s Pioneering Journey: Transforming Rare Disease Treatment Through Innovative Gene Therapy Part 1
As noted in a recent article published in Inside Precision Medicine, in 1992 gene therapy was often discussed but seldom practiced. When Jim Wilson MD, PhD attended Penn State, gene…
Continue Reading
Jim Wilson’s Pioneering Journey: Transforming Rare Disease Treatment Through Innovative Gene Therapy Part 1
- Go to the previous page
- 1
- …
- 3
- 4
- 5
- 6
- 7
- 8
- 9
- …
- 303
- Go to the next page
