Fragile X Syndrome
Biotech Firm Begins Phase IIa Trial for New Fragile X Therapy
In a recent statement, CONNECTA Therapeutics, a biotechnology company focused on disorders of the central nervous system, has announced the start of its Phase IIa
Diagnosing the Undiagnosed in Rare Disease: Worldwide, Multi-Institution, and Multi-disciplinary Work Underway
Opdivo–Cabometyx Combination Shows Significant Survival Benefit in Advanced Kidney Cancer
Supporting Siblings Through Loss: Remembering My Sister Heidi
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COVID-19
A Cumulative COVID-19 Report for April/May 2026
As seen in the CDC’s recent reports, 0.6 per 100,000 people were hospitalized in mid-April with confirmed hospital admissions averaging between 800 to 900 in
Thyroid eye disease
Phase 3 Trial Highlights Rapid, Sustained Benefit of Veligrotug in Thyroid Eye Disease
As reported on Medscape, global phase 3 clinical trial suggests that veligrotug, an investigational monoclonal antibody targeting the insulin-like growth factor‑1 receptor (IGF‑1R), may offer
Rare Disease
FDA Clarifies Marketing Limits for Compounded GLP-1 Drugs Amid Ongoing Enforcement Effort
As reported on Becker’s Hospital Review, the U.S. Food and Drug Administration (FDA) has issued updated guidance targeting how telehealth companies promote compounded medications, particularly
Diabetes Type 2
FDA Clears First Over-the-Counter Continuous Glucose Monitor for Children
As reported on CNET, parents and caregivers now have a new option to help monitor children’s blood sugar levels at home. The US Food and
Renal Cell Carcinoma
FDA Approves Pembrolizumab–Belzutifan Combination for Adjuvant Treatment of High-Risk Clear Cell RCC
As reported on Pharmaceutical Technology, the US Food and Drug Administration (FDA) has approved a new adjuvant treatment option for adults with clear cell renal
Rare Disease
FDA’s New Framework Signals Turning Point for Rare Disease Therapies—But Major Barriers Remain
As reported on MedCity News, the U.S. Food and Drug Administration (FDA) has introduced a new regulatory pathway that could reshape how treatments are developed
HIV/AIDS
FDA Reviews Gilead’s Once-Weekly Oral Lenacapavir for HIV Prevention
As reported on Business Wire, Gilead Sciences has announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application for
Lynch syndrome
Hereditary Cancer Risk Is Harder to Detect When Family History Crosses Borders
I grew up believing health was something you could control if you were disciplined enough. In my Chinese immigrant family, my father talked constantly about
Diabetes Type 1
FDA Grants Accelerated Approval to Teplizumab for Newly Diagnosed Pediatric Type 1 Diabetes
As shared in a recent press release, the U.S. Food and Drug Administration (FDA) has granted accelerated approval to teplizumab (Tzield) for a new pediatric
Idiopathic Hypersomnia
Alkermes’ Alixorexton Gains Orphan Drug Status in the US and EU
As reported by Pharmaceutical Technology, Alkermes has announced that its investigational therapy alixorexton has received orphan drug designation from both US and European regulators, marking
IgA Nephropathy
AstraZeneca’s Ravulizumab Receives FDA Priority Review for IgA Nephropathy
As reported on PharmaBiz, AstraZeneca has announced that the US Food and Drug Administration (FDA) has accepted for review a supplemental Biologics License Application (sBLA)
Rare Disease
FDA Signals Shift Toward Bayesian Methods in Clinical Trials
As reported on MedCity News, the U.S. Food and Drug Administration (FDA) has issued draft guidance encouraging the thoughtful use of Bayesian statistical approaches in
Thyroid eye disease
Glycemic Monitoring Recommended for All Patients Receiving Teprotumumab in Thyroid Eye Disease
Emerging evidence, as reported by the American Academy of Ophthalmology, suggests that glycemic surveillance should be considered for all patients undergoing teprotumumab therapy for thyroid
Rare Disease
Aortic Valvotomy, A Widening of Heart Valves
When Seth Wharton was only one month old, doctors repaired his narrow heart valve. Then, five years later, he was referred to UAB Cardiovascular Institute
Dravet Syndrome
An Update from the Dravet Syndrome Foundation
Madrid, June 5th, 2026 The Dravet Syndrome Foundation received the Merck Innovation Talent Award for a scientific publication regarding a tool that assesses neuropsychiatric issues
Neurotrophic Keratopathy
TearSolutions Advances Lacripep® for Neurotrophic Keratitis with FDA Designations and Phase 2 Trial Launch
As reported on Business Wire, TearSolutions, a clinical-stage biotechnology company focused on ophthalmic therapies, has announced key regulatory and clinical progress for its investigational drug
Fabry Disease
Fabry International Network’s Young Adult Guidebook
Website: https://fabrynetwork.org/youngadults Instagram Young Adults: @fabryyoungadults Instagram FIN: @fabryintnetwork 11 June 2026 – FIN launches international Young Adults Guidebook created by and for young adults
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People often see the appointments.
What they don't always see is everything that comes with them.
The scheduling. The rescheduling. The insurance calls. The paperwork. The reminders. The pharmacy pickups. The waiting on hold.
Sometimes managing the logistics can take as much energy as managing the condition itself.
If this part feels exhausting, you're not imagining it—and you're definitely not alone.
#chronicillness #InvisibleIllness #raredisease #youarenotalone #PatientWorthy
The “wobbles.”
It’s one of the first things many people notice about children with Ataxia-Telangiectasia (A-T).
They sway when they stand. They stumble when they walk. They wiggle. They shift. They move constantly.
Charlotte’s “wobbles” are part of her daily life and some days they’re more noticeable than others. What many people don’t realize is that for children with A-T, staying still can be incredibly difficult. It’s exhausting for them. Their brains are constantly working to coordinate movements that most of us never have to think about. Even something as simple as standing in one place, sitting upright, or waiting for your turn during swim lessons takes incredible effort.
The wobbles may be one of the visible signs of A-T but Charlotte doesn’t let the wobbles stop her from jumping in the water and trying new things. For now, they’re just part of how she moves through the world.
Children with A-T need a future where the wobbles won’t get worse, or actually where they don’t start at all.
#charlottesstory #charlottescure #ataxiatelangiectasia #atchildrensproject #cure
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