
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

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

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

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

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

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

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

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

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

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

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

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

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)

As reported on MedCity News, the U.S. Food and Drug Administration (FDA) has issued draft guidance encouraging the thoughtful use of Bayesian statistical approaches in

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

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

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

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

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
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
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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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Tom Seaman ... See MoreSee Less
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