Beta Thalassemia Major: This Couple Had a Baby to Save Their Son
source: pixabay.com

Beta Thalassemia Major: This Couple Had a Baby to Save Their Son

According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…

Continue Reading Beta Thalassemia Major: This Couple Had a Baby to Save Their Son
Experimental Therapies for Small Cell Lung Cancer and Acute Myeloid Leukemia Earn Orphan Drug Designation
source: pixabay.com

Experimental Therapies for Small Cell Lung Cancer and Acute Myeloid Leukemia Earn Orphan Drug Designation

According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug…

Continue Reading Experimental Therapies for Small Cell Lung Cancer and Acute Myeloid Leukemia Earn Orphan Drug Designation
Collaborative Study Highlights the Importance of Proteinuria in Focal Segmental Glomerulosclerosis
source: pixabay.com

Collaborative Study Highlights the Importance of Proteinuria in Focal Segmental Glomerulosclerosis

According to a story from BioSpace, a recent study conducted by the biotechnology company Goldfinch Bio in collaboration with academic partners is highlighting the importance of proteinuria in determining outcomes…

Continue Reading Collaborative Study Highlights the Importance of Proteinuria in Focal Segmental Glomerulosclerosis
RDLA Webinar Provides Vital Updates on Legislation Relevant to the Rare Disease Community
source: pixabay.com

RDLA Webinar Provides Vital Updates on Legislation Relevant to the Rare Disease Community

On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease…

Continue Reading RDLA Webinar Provides Vital Updates on Legislation Relevant to the Rare Disease Community
Experimental Gene Therapy for GM1 Gangliosidosis Earns Rare Pediatric Disease Designation
source: pixabay.com

Experimental Gene Therapy for GM1 Gangliosidosis Earns Rare Pediatric Disease Designation

According to a story from BioSpace, the gene therapy company Axovant Gene Therapies Ltd. recently announced that it has been given Rare Pediatric Disease designation from the US Food and…

Continue Reading Experimental Gene Therapy for GM1 Gangliosidosis Earns Rare Pediatric Disease Designation
FDA Grants IDE to Test Experimental Device for Pulmonary Arterial Hypertension
source: pixabay.com

FDA Grants IDE to Test Experimental Device for Pulmonary Arterial Hypertension

According to a story from GlobeNewswire, the medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…

Continue Reading FDA Grants IDE to Test Experimental Device for Pulmonary Arterial Hypertension
New Ultomiris Formulation Gets Approved for PNH and AHUS
source: pixabay.com

New Ultomiris Formulation Gets Approved for PNH and AHUS

According to a story from Market Watch, the biopharmaceutical company Alexion Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has approved a new formulation of…

Continue Reading New Ultomiris Formulation Gets Approved for PNH and AHUS
Waldenström’s Macroglobulinemia Precursor States: Could They be Key to a Cure?
source: pixabay.com

Waldenström’s Macroglobulinemia Precursor States: Could They be Key to a Cure?

On October 14th, 2020, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar featuring Dr. Irene Ghobrial of the Dana Farber Cancer Institute. The focus of her presentation was on…

Continue Reading Waldenström’s Macroglobulinemia Precursor States: Could They be Key to a Cure?
Trodelvy Gets Orphan Drug Designation as a Glioblastoma Treatment
source: pixabay.com

Trodelvy Gets Orphan Drug Designation as a Glioblastoma Treatment

According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the…

Continue Reading Trodelvy Gets Orphan Drug Designation as a Glioblastoma Treatment
Gene Therapy Receives Rare Pediatric Disease Designation for Sandhoff Disease and Tay-Sachs Disease
source: pixabay.com

Gene Therapy Receives Rare Pediatric Disease Designation for Sandhoff Disease and Tay-Sachs Disease

According to a story from GlobeNewswire, the gene therapy company Axovant Gene Therapies Ltd. recently announced that its gene therapy AXO-AAV-GM2 has been given Rare Pediatric Disease designation by the…

Continue Reading Gene Therapy Receives Rare Pediatric Disease Designation for Sandhoff Disease and Tay-Sachs Disease
The Glanzmann’s Research Foundation: Dedicated to Finding a Cure
source: pixabay.com

The Glanzmann’s Research Foundation: Dedicated to Finding a Cure

Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…

Continue Reading The Glanzmann’s Research Foundation: Dedicated to Finding a Cure
Experimental WHIM Syndrome Treatment Gets FDA Fast Track Designation
source: pixabay.com

Experimental WHIM Syndrome Treatment Gets FDA Fast Track Designation

According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…

Continue Reading Experimental WHIM Syndrome Treatment Gets FDA Fast Track Designation
Zeposia as a Treatment for Ulcerative Colitis Shows Positive Results in Phase 3 Trial
source: pixabay.com

Zeposia as a Treatment for Ulcerative Colitis Shows Positive Results in Phase 3 Trial

According to a story from Pharma Advancement, Bristol Myers Squibb recently announced encouraging findings from a phase 3 clinical trial. This study was testing its drug ozanimod (marketed as Zeposia)…

Continue Reading Zeposia as a Treatment for Ulcerative Colitis Shows Positive Results in Phase 3 Trial
Duchenne Muscular Dystrophy Gene Therapy Gets FDA Fast Track Designation
source: pixabay.com

Duchenne Muscular Dystrophy Gene Therapy Gets FDA Fast Track Designation

According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…

Continue Reading Duchenne Muscular Dystrophy Gene Therapy Gets FDA Fast Track Designation
Massachusetts General Cancer Center Receives the First Incyte Ingenuity Award for GvHD
source: pixabay.com

Massachusetts General Cancer Center Receives the First Incyte Ingenuity Award for GvHD

The global biopharmaceutical company Incyte recently announced the first-ever recipient of its Incyte Ingenuity Award, which will be the Massachusetts General Cancer Center (MGCC). The purpose of this award is…

Continue Reading Massachusetts General Cancer Center Receives the First Incyte Ingenuity Award for GvHD
Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding
source: pixabay.com

Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding

According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…

Continue Reading Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding
Experimental Renal Cell Carcinoma Combination Treatment Shows Promise in Early Trial
source: pixabay.com

Experimental Renal Cell Carcinoma Combination Treatment Shows Promise in Early Trial

According to a story from Targeted Oncology, an experimental combination therapy tested on patients with advanced clear cell renal cell carcinoma (ccRCC) appeared to show potential for efficacy in a…

Continue Reading Experimental Renal Cell Carcinoma Combination Treatment Shows Promise in Early Trial
American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign
source: pixabay.com

American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign

According to a press release from the American Kidney Fund (AKF), the organization, which is dedicated to fighting kidney disease, has partnered with the biotech company Sanofi Genzyme in a…

Continue Reading American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign
Can AI and Machine Learning Help Rare Patients Get Diagnosed and Treated Faster?
source: pixabay.com

Can AI and Machine Learning Help Rare Patients Get Diagnosed and Treated Faster?

A research team from IPM.ai, a subsidiary of Swoop, and Insmed, a biotechnology company, recently collaborated on a case study that demonstrated how AI and machine learning could be used…

Continue Reading Can AI and Machine Learning Help Rare Patients Get Diagnosed and Treated Faster?
Close Menu