medical research – Page 9

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Rare Community Profiles: Study Investigator Dr. Weyand Discusses the Trial Data that Led to ALTUVIIIO Approval for Hemophilia A

Post author:Jessica Lynn
Post published:April 25, 2023
Post category:Hemophilia A

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

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Phase 2a Study on ‘1104 in Allergic Disease Now Complete

Post author:Jessica Lynn
Post published:April 18, 2023
Post category:Eosinophilic Esophagitis

Revolo Biotherapeutics was founded with one specific goal in mind: to benefit people living with autoimmune and allergic diseases by creating novel, life-changing therapeutics that induce long-term remission. They…

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How Preoperative Immunotherapy Can Change the MPM Treatment Landscape and Improve Outcomes

Post author:Jessica Lynn
Post published:April 17, 2023
Post category:Malignant pleural mesothelioma Mesothelioma

Historically, mesothelioma has been difficult to treat. Although treatment options exist, the cancer tends to respond poorly which results in a higher mortality rate. Improving patient care and outcomes (such…

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A Biomarker for Niemann-Pick Disease Type C Could Improve Congenital Disorders of Glycosylation Diagnosis

Post author:Jessica Lynn
Post published:April 17, 2023
Post category:Congenital disorders of glycosylation Niemann-Pick Disease Niemann-Pick Type C Disease

Let’s start with a little vocabulary lesson. Today’s word? Biomarker. A biological marker, or biomarker, refers to some sort of measurable indicator of disease. For example, blood pressure, cellular gene…

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Researchers Work to Develop Novel Gene Therapies for Alport Syndrome

Post author:Jessica Lynn
Post published:April 14, 2023
Post category:Alport syndrome

Medical research is crucial in better understanding diseases and discovering novel treatment options. Unfortunately, there is often one large barrier to research: funding. This is especially true within the rare…

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Mapping the Immune Response to P. Falciparum Explains How Malaria Evades Treatment

Post author:Jessica Lynn
Post published:April 13, 2023
Post category:Malaria

Over the years, scientists have learned that Plasmodium falciparum (P. falciparum) is tricky. This parasite, which causes the deadliest type of malaria in humans (falciparum malaria), is transmitted through the…

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Enrollment Complete for Phase 3 Study Evaluating Brensocatib for Non-CF Bronchiectasis

Post author:Jessica Lynn
Post published:April 12, 2023
Post category:Bronchiectasis

People living with bronchiectasis, a chronic condition characterized by damaged airways, experience progressive lung damage and inflammation. This can lead to a multitude of consequences, including a reduction in…

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First Patient Dosed in Phase 1 Study to Evaluate QRL-201 for ALS

Post author:Jessica Lynn
Post published:April 12, 2023
Post category:Amyotrophic Lateral Sclerosis

STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research…

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Pitolisant Reduced Excessive Daytime Sleepiness in Children with Narcolepsy

Post author:Jessica Lynn
Post published:April 11, 2023
Post category:Narcolepsy

Currently, the standards-of-care for narcolepsy include lifestyle changes involving diet and exercise, behavioral therapy, nap therapy, and certain medications designed to combat excessive daytime sleepiness. However, a majority of…

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Pembrolizumab Monotherapy Confers Benefits for Advanced Thyroid Carcinoma

Post author:Jessica Lynn
Post published:April 11, 2023
Post category:Thyroid Cancer

Data from the first cohort of the Phase 2 KEYNOTE-158 trial, evaluating pembrolizumab for individuals with advanced thyroid carcinoma, showed positive safety and efficacy. According to Cancer Network, 103 participants…

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Scientists Identify Biomarkers to Predict Pancreatic Cancer Development

Post author:Jessica Lynn
Post published:April 10, 2023
Post category:Pancreatic Cancer

Cases of pancreatic cancer have been increasing throughout the United States over the past few decades. Although the 5-year survival rate recently rose to 12%, pancreatic cancer is on track…

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Family Raises Funds for PDCD Research After Daughter’s Diagnosis

Post author:Jessica Lynn
Post published:April 7, 2023
Post category:Pyruvate dehydrogenase deficiency

The Higbee family never planned to become rare disease advocates. But when their daughter Harlow began experiencing health issues, they knew that they would do whatever they could to help.…

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Mevalonolactone Treatment Eased Symptoms of Statin Myopathy and a form of LGMD

Post author:Jessica Lynn
Post published:April 7, 2023
Post category:Limb-girdle muscular dystrophy

Statins are a type of drug that can help you to lower your blood cholesterol levels. These drugs work by inhibiting HMG CoA reductase, an enzyme that the body…

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ATRIP Gene Variants Could Increase Breast Cancer Susceptibility

Post author:Jessica Lynn
Post published:April 6, 2023
Post category:Breast Cancer

In the past, there have been specific genetic mutations associated with breast cancer: most often the BRCA1 and BRCA2 genes. Altogether, there are 15 genes associated with a moderate-to-high…

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Cyclosporine Contributes to Long-Term Treatment Efficacy in Ulcerative Colitis (UC)

Post author:Jessica Lynn
Post published:April 6, 2023
Post category:ulcerative Colitis

Certain patients with ulcerative colitis (UC) do not respond well to steroid treatment. These patients are considered to have steroid-refractory acute severe ulcerative colitis, or ASUC. Other therapeutic options, such…

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Emicizumab is an Effective Prophylaxis for Hemophilia A, Study Shows

Post author:Jessica Lynn
Post published:April 6, 2023
Post category:Hemophilia Hemophilia A

Approved for use in 2017, emicizumab—marketed under the brand name Hemlibra—has shown immense benefits in the treatment of hemophilia A. But researchers wanted to better understand how emicizumab, used…

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MDA Announces Research Grants for Duchenne Muscular Dystrophy

Post author:Jessica Lynn
Post published:April 4, 2023
Post category:Duchenne Muscular Dystrophy

Medical research is incredibly important, especially within the rare disease community. Rare conditions tend to be under-researched and under-funded; this can make it difficult not only to better learn…

Atrial Fibrillation May Be Significantly Underdiagnosed

Post author:Jessica Lynn
Post published:April 4, 2023
Post category:Atrial Fibrillation

What is the actual prevalence of atrial fibrillation (AF) on a local, national, and global scale? While this question may seem straightforward, the answer is not. An estimated 33% of…

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UPMC Launches TANGO2 Research to Help Families

Post author:Jessica Lynn
Post published:March 30, 2023
Post category:TANGO2 disease

A mother knows best. In Kaitlyn Fryar's case, her intuition about her son Hudson led her to pursue help from numerous doctors. According to CBS Pittsburgh, Hudson seemed to…

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Rare Community Profiles: How the #RAREis Global Advocate Grant Supported the E. WE Foundation: A Discussion with Sarita Edwards

Post author:Jessica Lynn
Post published:March 29, 2023
Post category:Rare Disease Trisomy 18

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

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“Napa in Miami” Wine Tasting and Auction Raised $1.2M+ for DMD Research

Post author:Jessica Lynn
Post published:March 28, 2023
Post category:Duchenne Muscular Dystrophy

CureDuchenne, the nation’s leading nonprofit dedicated to funding a cure for Duchenne muscular dystrophy (DMD) and a Patient Worthy partner, held its first “Napa in Newport” event in 2015;…

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Deuruxolitinib Shows Promise for Alopecia Areata, Studies Show

Post author:Jessica Lynn
Post published:March 28, 2023
Post category:Alopecia areata

The American Academy of Dermatology (AAD) held its Annual Meeting from March 17-21, 2023. During the meeting, stakeholders from the dermatology community connected to discuss clinical practices, research trends, and…

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Axitinib-Avelumab Combination Prompted Responses in Recurrent or Metastatic Adenoid Cystic Carcinoma (ACC)

Post author:Jessica Lynn
Post published:March 27, 2023
Post category:Adenoid Cystic Carcinoma

While the five-year survival rate for adenoid cystic carcinoma (ACC) is relatively high, this cancer has a high risk of recurrence. Therapeutic options are needed to manage this cancer and…

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Cabometyx-Nivolumab Combo Improves Survival in Advanced RCC, Study Results Show

Post author:Jessica Lynn
Post published:March 27, 2023
Post category:Renal Cell Carcinoma

In early stages, renal cell carcinoma (RCC) is treatable and has a high five-year survival rate. However, if the cancer progresses or metastasizes (spreads), the five-year survival rate falls…

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CURE ID: Drug Repurposing Provides Big Impact for Patients

Post author:Patient Worthy Contributor
Post published:March 24, 2023
Post category:Rare Disease

Contributed by Critical Path Institute (C-Path) and republished by Patient Worthy - find the original source article here. C-Path’s CDRC is positioned to inform future clinical trials for diseases of…

Rare Community Profiles: Study Investigator Dr. Weyand Discusses the Trial Data that Led to ALTUVIIIO Approval for Hemophilia A

Phase 2a Study on ‘1104 in Allergic Disease Now Complete

How Preoperative Immunotherapy Can Change the MPM Treatment Landscape and Improve Outcomes

A Biomarker for Niemann-Pick Disease Type C Could Improve Congenital Disorders of Glycosylation Diagnosis

Researchers Work to Develop Novel Gene Therapies for Alport Syndrome

Mapping the Immune Response to P. Falciparum Explains How Malaria Evades Treatment

Enrollment Complete for Phase 3 Study Evaluating Brensocatib for Non-CF Bronchiectasis

First Patient Dosed in Phase 1 Study to Evaluate QRL-201 for ALS

Pitolisant Reduced Excessive Daytime Sleepiness in Children with Narcolepsy

Pembrolizumab Monotherapy Confers Benefits for Advanced Thyroid Carcinoma

Scientists Identify Biomarkers to Predict Pancreatic Cancer Development

Family Raises Funds for PDCD Research After Daughter’s Diagnosis

Mevalonolactone Treatment Eased Symptoms of Statin Myopathy and a form of LGMD

ATRIP Gene Variants Could Increase Breast Cancer Susceptibility

Cyclosporine Contributes to Long-Term Treatment Efficacy in Ulcerative Colitis (UC)

Emicizumab is an Effective Prophylaxis for Hemophilia A, Study Shows

MDA Announces Research Grants for Duchenne Muscular Dystrophy

Atrial Fibrillation May Be Significantly Underdiagnosed

UPMC Launches TANGO2 Research to Help Families

Rare Community Profiles: How the #RAREis Global Advocate Grant Supported the E. WE Foundation: A Discussion with Sarita Edwards

“Napa in Miami” Wine Tasting and Auction Raised $1.2M+ for DMD Research

Deuruxolitinib Shows Promise for Alopecia Areata, Studies Show

Axitinib-Avelumab Combination Prompted Responses in Recurrent or Metastatic Adenoid Cystic Carcinoma (ACC)

Cabometyx-Nivolumab Combo Improves Survival in Advanced RCC, Study Results Show

CURE ID: Drug Repurposing Provides Big Impact for Patients

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