As reported on MedicalXpress, researchers have reported that urolithin A, a compound derived from gut metabolism and available as an over-the-counter supplement, enhanced the effectiveness
Today the Tin Soldier Disease has a treatment. A clinical trial showed a 90% reduction in new bone development in adults with FOP. After Decades
Researchers Discover That One of Medicine’s Most Common Lab Tests May Warn of Rare But Serious Side Effects Weeks Before They Occur As reported on
As reported on Fierce BioTech, the U.S. Food and Drug Administration has granted approval to Lantheus’ positron emission tomography (PET) imaging agent Tauklarify (MK-6240), expanding
Scientists Discover Multiple Pathways Through Which an Ancient Fruit Protects the Cardiovascular System For centuries, pomegranates have held a special place in Middle Eastern and
Living with dystonia and other movement disorders means navigating far more than muscle spasms or abnormal postures—and if have dystonia, you already know this. As patients,
The Basic Problem: Why One Bad Gene Can Cause Big Trouble Imagine your body as a giant factory. Like any factory, it needs quality control—machines
As reported on BioSpace, the U.S. Food and Drug Administration has approved Regeneron’s Pasatru (garetosmab) for adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic
Historic Milestone Offers Relief from Relentless Burden of Glycogen Storage Disease Type Ia According to a recent press release from Ultragenyx, after decades of medical
As reported by allsci, China’s National Medical Products Administration (NMPA) has granted conditional approval to TransThera Sciences’ Yochanra (tinengotinib) for adults with unresectable, advanced, or
As reported by PharmaBiz, Zydus Lifesciences has announced the launch of Revahale (revefenacin), a once-daily nebulized long-acting muscarinic antagonist (LAMA) for the maintenance treatment of
What Happened? Hansa Biopharma, a Swedish company, announced that the first patient in Australia has successfully received a kidney transplant after being treated with a
As reported on BioSpace, AstraZeneca has discontinued a pivotal Phase 3 trial evaluating its investigational bispecific antibody volrustomig in advanced non-small cell lung cancer (NSCLC)
New observational research suggests that addressing obesity alongside inflammatory joint disease could improve treatment outcomes for people with psoriatic arthritis (PsA). As reported on MedIndia,
As reported on News Medical, researchers are highlighting a new class of therapies that could improve outcomes for people living with primary biliary cholangitis (PBC),
Researchers at MIT have identified a potential new approach to reducing lung cancer risk by targeting caspase-1, an enzyme involved in inflammation. Findings published in
A recent study shared by The Lancet shows that a first-in-class investigational therapy targeting neurotrophin-3 (NT-3) significantly reduced pain and improved function in patients with
As reported by the ICIJ, the FDA originally approved Keytruda (pembrolizumab) combined with cisplatin and gemcitabine on October 31, 2023, to treat locally advanced inoperable
CureDuchenne #GeneEditing @PrecisionBiosciencesPrecision BioSciences has dosed the first individual with their novel gene editing approach for individuals with Duchenne mutations between exons 45 and 55
Precision BioSciences has shared that the first participant in the FUNCTION_DMD clinical trial has received their investigational gene editing treatment, PBGENE-DMD. PBGENE-DMD delivers ARCUS nucleases via AAV9 to cut out exons 45-55 of the dystrophin gene, a therapeutic approach amenable to individuals with Duchenne with mutations in that region.
Link to press release and community letter: cureduchenne.org/research/precision-biosciences-has-dosed-the-first-individual-with-their-novel-g...
#DMD #duchennemusculardystrophy #GeneEditing
Cure GM1 Foundation #backtoschool #rarediseaseIt is back to school season, and every family's story looks a little different. For families navigating rare disease, the new school year often means extra preparation, extra advocacy, and extra love. To every GM1 parent and caregiver getting ready for this season, we are cheering for you. 💚
#BackToSchool #CureGM1
Amyloidosis Foundation TOMORROW!✨TOMORROW'S SESSION ✨
Wednesday, August 26, 6:00 PM – 7:00 PM America/New_York
Learn how cardiac testing can help detect and monitor heart involvement in amyloidosis. We will explain common tests, what to expect, and how results may guide care, with time for patient and caregiver questions.
Dr. Hasan Siddiqi, MD, MSCR, FACC
Hasan Siddiqi MD, MSCR, FACC is an advanced heart failure cardiologist and directs its cardiac amyloidosis program. He manages amyloidosis, heart failure, VAD, and transplant patients in the world's largest heart transplant center, leads research and trials, and trained at Harvard, Michigan, Penn, and Brigham.
To Register: us06web.zoom.us/meeting/register/AZDwB7PuTKmWDWoW1P_Dzg
Zoom Meeting ID: 81845030592
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