Rare Disease
Genomics England World Leading Research Study
The Genomics England Research Study listed approximately two hundred rare treatable conditions of children five years old or younger. Genomics has had, and continues to
More Than 1,000 Patients Enrolled in Major UK Trial Testing One-Week Breast Cancer Radiotherapy
Advancing Precision Oncology: Digital Twins and Symbolic AI in Triple-Negative Breast Cancer
Open-Source Image Analysis Tool Provides Quantitative Assessment of MASH in Mouse Models
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THE LATEST FROM PATIENT WORTHY
Ichthyosis
Living in My Skin: A Young Man’s Life with Ichthyosis
My name is Sami Yusuf Baker. I am 19 years old, and I live in a small village in the Kurdistan Region of Iraq. I
Pulmonary veno-occlusive disease
I Am Rare, I Am Here
My name is Rim Wafaa Bendjaada. I am 22 years old, a rare disease and pulmonary hypertension advocate from Algeria, living with pulmonary veno-occlusive disease
Rare Disease
All the Warm Blankets
Editor’s Note: The following is an excerpt from Bird’s Eye View: Stories of a Life Lived in Health Care by Sue Robins. I remember every single
Metabolic dysfunction-associated steatohepatitis
Spatial Mapping Study Uncovers Distinct Molecular Changes Across Liver Regions in MASH
As reported on Bioengineer and ScienMag, a new spatial transcriptomics study has provided fresh insight into how different regions of the liver are remodeled during
Cervical Dystonia
The Struggle Beneath the Struggle: Letting Go of Resistance
When life changes in a way we never wanted or expected, there are often two struggles taking place: the hardship itself and our struggle against
Breast Cancer
Personalizing Endocrine Therapy Plans: Help Patients with Early-Stage, HR+ Breast Cancer Find Clarity About What’s Next
Editor’s Note: The following article was originally written and shared with us by Adriana Olivo, NP, Memorial Sloan Kettering Cancer Center. For many people with
Fibrodysplasia Ossificans Progressiva (FOP)
The Road to Diagnosis – Holly’s FOP Story
Editor’s Note: The following story was originally written and shared with us by Holly M. LaPrade. Thirty years ago, a small army of doctors cautiously
Clear cell renal cell carcinoma
New Molecular Scan Shows Promise for Mapping Clear Cell Kidney Cancer
Clear cell renal cell carcinoma (ccRCC) is the most common type of kidney cancer. It begins in the kidney’s filtering cells and gets its name
Rare Disease
Alentis Deepens ADC Strategy Through New Claudin-1 Collaboration With PinotBio
As reported on Pharmaceutical Technology, Alentis Therapeutics has expanded its efforts in claudin-1-targeted cancer therapeutics through a newly announced partnership with PinotBio, aimed at generating
Cancer
Danish Study Examines Childhood Cancer After Fertility Treatment
As reported on Inside Precision Medicine, a large study from Denmark found that children conceived with certain assisted reproductive technologies had a higher relative rate
IgA Nephropathy
Otsuka’s Phase 3 VISIONARY Study Suggests Voyxact May Alter Disease Course in IgA Nephropathy
Final two-year data from the Phase 3 VISIONARY trial indicate that Voyxact (sibeprenlimab-szsi) may significantly slow progression of primary immunoglobulin A nephropathy (IgAN), preserving kidney
Rare Disease
Reinvigorating the Biological Clock: How a Diabetes Target Could Help Combat Ovarian Aging
One of the most persistent hurdles in biology is the natural, age-related decline of female fertility. As women age, both the quantity and quality of
Myasthenia Gravis
The Space Between Appointments
Patient by Diagnosis. Founder by Decision. By Camden Hill, Founder & CEO of Areluno Health I was 20 years old when my body started quitting
Rare Disease
Many Nondrug Treatments for Low Back Pain May Offer Good Value, Review Finds
Exercise, yoga, psychological support and other approaches that do not involve medication or surgery may help people with low back pain—and may be cost-effective, according
Colorectal cancer
Alliance Announces FDA Approval of Atezolizumab Based on Results from the Phase III ATOMIC Trial for Patients with Stage III dMMR Colon Cancer
Editor’s Note: This article, reprinted in full, was originally written and shared with us by Alliance. To see the article in its original format, please
Hunter Syndrome
Jay’s Hunter Syndrome Story
All genetic testing we did for Jay during pregnancy came back normal (though we did the bare minimum). When he was born, he struggled to
C. Diff
Experimental Treatment Targets C. diff While Sparing More of the Gut’s Microbes
A C. diff infection can turn a hospital stay into a much harder experience. The bacterium can cause severe diarrhea, abdominal pain and inflammation of
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Pitt Hopkins Research FoundationIF WE DON'T SHOW UP, why should anyone else?
PARENTS! If you want the FDA to show up for our kids, we need you to show up on NOV. 13!!
Register today! us06web.zoom.us/.../register/6lKBS5t4QbWpBdFq6KOH-Q
More details at pitthopkins.org/pfddmeeting/.
Cure GRIN FoundationThank you to everyone who participated in our Healthcare Assessment! In collaboration with the Grin2b Foundation, Cacna1a Foundation, and CAMK2 Therapeutics Network, we set out to learn what care currently looks like for people with these rare neurodevelopmental disorders, and where the biggest challenges and gaps exist. We received an incredible 244 responses from the GRI community!
Together with the GRIN2B Foundation, we’re sharing the data collected specifically from GRI families in the infographic below.
The results highlight important challenges, including limited access to specialists, gaps in treatment, and the burden on families to coordinate care across providers.
Thank you to all the GRI families who shared their experiences and helped shine a light on these important healthcare needs!
