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A Clinical Trial of Gemcabene in Paediatric Patients with Non-Alcoholic Fatty Liver Disease Has Been Stopped Following Safety Concerns

A Clinical Trial of Gemcabene in Paediatric Patients with Non-Alcoholic Fatty Liver Disease Has Been Stopped Following Safety Concerns

Gemphire Therapeutics Inc. has been recommended to stop a clinical trial by the Data and Safety Monitoring Board at Emory University School of Medicine. The clinical trial, which ran into…

Continue Reading A Clinical Trial of Gemcabene in Paediatric Patients with Non-Alcoholic Fatty Liver Disease Has Been Stopped Following Safety Concerns
For the First Time in 30 Years: A New Advancement in Rhabdomyosarcoma Treatment

For the First Time in 30 Years: A New Advancement in Rhabdomyosarcoma Treatment

  According to MedPage Today, a phase III study showed that 6 additional months of chemotherapy for maintenance led to a sizable increase in the 5-year overall survival rate for…

Continue Reading For the First Time in 30 Years: A New Advancement in Rhabdomyosarcoma Treatment
Enrollment Begins in Duchenne Muscular Dystrophy Trial

Enrollment Begins in Duchenne Muscular Dystrophy Trial

According to a story from PR Newswire, the specialty drug development company Mallinckrodt recently announced that enrollment for trials of its experimental medication MNK-1411 has started. The drug will be…

Continue Reading Enrollment Begins in Duchenne Muscular Dystrophy Trial
Research on Treating Mice for Gaucher Disease in Utero Offers Hope

Research on Treating Mice for Gaucher Disease in Utero Offers Hope

According to a recent article for myscience.org, there is new potential for genetic therapy to cure the often deadly neurodegenerative disorder known as Gaucher disease. The new studies were first…

Continue Reading Research on Treating Mice for Gaucher Disease in Utero Offers Hope
Trial Data Reveals Effectiveness of Investigational Treatment for Ulcerative Colitis

Trial Data Reveals Effectiveness of Investigational Treatment for Ulcerative Colitis

According to a story from the pharmaceutical company Takeda, the company recently released the results from its clinical trial testing an experimental subcutaneous formula of the drug vedolizumab. The drug…

Continue Reading Trial Data Reveals Effectiveness of Investigational Treatment for Ulcerative Colitis
Results From Studies of an Experimental Drug for Chronic Kidney Disease Have Been Announced

Results From Studies of an Experimental Drug for Chronic Kidney Disease Have Been Announced

Results from two studies looking at the effects of an experimental drug (bardoxolone) on kidney function in patients with chronic kidney disease (CKD) have been released. One of the studies…

Continue Reading Results From Studies of an Experimental Drug for Chronic Kidney Disease Have Been Announced
Editor’s Choice: Investigating Anxiety and Invisible Pain with Rare Disease

Editor’s Choice: Investigating Anxiety and Invisible Pain with Rare Disease

Happy End-of-July! As the week comes to a close, we want to spotlight four articles. We have an honest piece from PW contributor, Tom Seaman. Next, we investigate the root…

Continue Reading Editor’s Choice: Investigating Anxiety and Invisible Pain with Rare Disease
Treating Fetuses With Gene Therapy Could Minimize the Damage From Genetic Disorders Like Gaucher Disease

Treating Fetuses With Gene Therapy Could Minimize the Damage From Genetic Disorders Like Gaucher Disease

According to a story from nature.com, gene therapy administered to fetuses could help curtail some of the worst effects of deadly genetic disorders. In a mouse model study, fetal delivery…

Continue Reading Treating Fetuses With Gene Therapy Could Minimize the Damage From Genetic Disorders Like Gaucher Disease
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You Are Not Alone: Empowering the Advanced Kidney Cancer Community
Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
*cancer that cannot be removed with surgery or has spread to other parts of the body. GEJ= gastroesophageal junction (where the esophagus joins the stomach)
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