PatientSight

Congratulations to Vera Therapeutics, Inc. and other life science companies who are bringing new treatments for IgA Nephropathy to market! It will be important to assess and measure IgAN patient experience to ease the path toward initiating and sustaining effective treatment in this increasingly competitive therapeutic area. #IgANephropathy #PatientExperience

I am excited to share that PatientSight is beginning to roll out the life science industry's first patient experience assessment tool that covers the whole healthcare journey. Patient experience benchmarking is common practice with health systems, but this is not the case in life sciences. This report goes well beyond internal customer satisfaction measurements. To learn more, please visit: https://lnkd.in/gxVQTrge #PatientExperience #PatientCentricity

June 20th is #WorldFSHDDay. For this occasion, I was fortunate to get life science executive and FSHD father Raymond A. Huml to write an article on this #raredisease as only he can do. It is full of keen knowledge and personal experience, leaving us with some hope for better treatments. To read Ray's excellent piece, please go to: https://lnkd.in/gjRqVN5u

Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disease characterized by fluctuating muscle weakness and fatigue that can affect the eyes, face, swallowing, speech, breathing, and limb movements. In recognition of June being MG Awareness Month, PatientSight has published survey findings asking MG patients what they most want life science companies to know about living with this condition. #gMG #myastheniagravis #MGAwarenessMonth #raredisease Read the summary here: https://lnkd.in/g4F39nUv

More than 80% of people living with generalized myasthenia gravis are AChR antibody positive. Treatment options exist for those who fall outside this category but are more limited. MuSK antibody positive gMG patients can often have more severe symptoms, which include difficulty with speech and swallowing, facial and neck muscle weakness, and respiratory involvement. Fortunately, more treatments are now available for this less common sub-type of gMG. Our research shows that the feature MuSK antibody positive gMG patients hope for the most is being able to be treated at home. #myastheniagravis

May is National #cysticfibrosis month. CF patients have benefited tremendously from a number of new treatments, but PatientSight research shows that 59% of 30 CF respondents were not totally satisfied with their treatment, showing much remaining unmet need. Vertex clearly has dominated this space, but there are several other companies developing products for CF using new technologies such as mRNA and gene therapy. Vertex Pharmaceuticals Sionna Therapeutics ReCode Therapeutics Krystal Biotech, Inc.

In recognition of ALS Awareness Month, I am sharing some research findings. As part of PatientSight's Rare Disease Patient Tracker, we asked 20 ALS patient and caregivers about living with the condition. Despite the extreme challenges described, many do still maintain a positive outlook for simple joys they still have. For the summary of this open-ended question, go to: https://lnkd.in/esQ_JhZQ #ALS

Congratulations to UCB and Neurona Therapeutics on this acquisition, which will help bring the promise of regenerative cell therapy to epilepsy patients! I've been posting about cell and gene therapy lately, and this is another example of the broadening scope of this treatment approach.

With Eli Lilly and Company's acquisition of Kelonia Therapeutics and their novel in vivo CAR T-cell therapy, it got me thinking about where things are in cell & gene therapy and where this treatment approach is heading. PatientSight included C&GT in a rare disease survey last year, so this can be considered a "baseline". What is encouraging is that three-quarters of those with experience in C&GT rate it quite highly. With more patient-friendly improvements and education about it, I see C&GT having a bright future.

Potential game-changer in CAR-T! #celltherapy #multiplemyeloma Kelonia Therapeutics https://lnkd.in/e3G_-Ugj