CheckOrphan is the leading #news and information platform dedicated to #rarediseases and #orphandrugs
Santa Cruz, USA / Basel, CH
Joined April 2009
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Rare Diseases retweeted
The @US_FDA grants Fast Track Designation (FTD) to $PLYX's PLX-200 for the treatment of CLN3 Disease, Krabbe disease & Sandhoff disease. PLX-200 now holds FTD for all prospective indications for evaluation in the SOTERIA Phase 2 basket trial.
Read more: bit.ly/4dVKL7Z
@CheckOrphan @KrabbeConnect @NTSAD @rarediseaseday
Rare Diseases retweeted
@CheckOrphan Exciting news for families affected by DMD: a new steroid alternative shows promise in helping boys with Duchenne maintain growth! This could be a game changer for many. Read more about this hopeful development here: buff.ly/EtbLMrk
Rare Diseases retweeted
#PolaryxTherapeutics joins the global #raredisease community in raising awareness and supporting patients, families, caregivers, and healthcare providers.
Full press release here: bit.ly/4cP9KZU
#RareDiseaseDay @CheckOrphan @rarediseaseday
#LysosomalStorageDisorders
Rare Diseases retweeted
@raremag @STEMUKPROJECT @eduSTEMUK @RareRevolutionM @raredisorders @CheckOrphan @rarediseaseday
Places are filling up for Genetics Matters Rare Disease Day event on 28/02 in Newcastle. Meet the scientists, hear about latest developments and show support for rare disease patients
Rare Diseases retweeted
Hi @CheckOrphan I’m working with researchers developing a clinical trial for people with T-Cell lymphoma. We’re keen to hear experiences of patients, carers & those who have lost loved ones. Pls can you share & encourage people to get in touch [email protected]
Rare Diseases retweeted
@CheckOrphan Exciting news for families affected by DMD! New data shows that DYNE-251 boosts dystrophin levels and helps preserve function, bringing hope for a better future. Learn more about these promising developments here: buff.ly/eE6wbu1
Rare Diseases retweeted
@CheckOrphan We're excited to share positive results from the recent Deramiocel trial in patients with DMD! This breakthrough brings hope for many families facing the challenges of Duchenne Muscular Dystrophy. Discover more about these promising outcomes here: buff.ly/IFengpe
Rare Diseases retweeted
@CheckOrphan Exciting news for families affected by DMD! Long-term data shows vamarolone is as effective as standard steroids but offers a safer alternative for our children. Let's keep pushing for better treatments! Read more here: buff.ly/YOv7MLm
Rare Diseases retweeted
@CheckOrphan Exciting research highlights how the muscle protein desmin may offer protection for the heart in those with DMD. This discovery could bring hope to families affected by Duchenne Muscular Dystrophy. Read more about this promising development here: buff.ly/YtCqgPq
Rare Diseases retweeted
@CheckOrphan Exciting advancements in DMD treatment! 🌟 A new article explores how microdystrophin gene therapy not only improves muscle function but also raises potential heart risks. It's a vital read for parents navigating this journey. Discover more: buff.ly/xhfUDHl
Rare Diseases retweeted
@CheckOrphan Discover the transformative power of eye gaze technology in our latest article, "My world through eye gaze technology." This innovation is changing lives for those with DMD, offering new ways to connect and communicate. Read more about this inspiring journey: buff.ly/T5FLhF3
Rare Diseases retweeted
@CheckOrphan New hope for families impacted by DMD! A new study reveals that trehalose may enhance microdystrophin therapy, potentially improving outcomes for our loved ones. Discover the latest advances in treatment. Read more here: buff.ly/i4Sx2uv
Rare Diseases retweeted
@CheckOrphan A new survey reveals that as DMD progresses, families are increasingly open to gene therapy options. This could be a turning point for many. Discover the insights that could shape the future of treatment: buff.ly/LhHy7YU
Rare Diseases retweeted
@CheckOrphan A new study reveals an early decline in trunk control among boys with DMD, highlighting the need for timely interventions. Understanding these changes can help families and caregivers better support their loved ones. Read more about this important research here: buff.ly/EPrnFOg
Rare Diseases retweeted
@CheckOrphan Discover the inspiring story of a Norwegian man's passion for gaming that led to a Netflix film and raised millions for DMD research. His journey shines a light on hope and innovation for families affected by Duchenne Muscular Dystrophy. Read more here: buff.ly/L6DjZ6J
Rare Diseases retweeted
Minovia Therapeutics announces positive interim results of the ongoing Phase 2 trial of the Company’s lead investigational compound MNV-201 in Pearson Syndrome, a fatal pediatric mitochondrial disease characterized by sideroblastic anemia (a disorder causing abnormal red blood cell development), failure to thrive and exocrine pancreas dysfunction.
Press Release: globenewswire.com/news-relea…
#MinoviaTherapeutics #MNV201 #PearsonSyndrome #MitochondrialDisease #RareDisease
Rare Diseases retweeted
This #CitizenScienceMonth, every rare disease story counts. 🧬💬 Join #OneMillionActsofScience by taking part in our Living Rare Study. Share your journey and help change the future of rare disease care.
👉 livingrarestudy.org
Thanks @SciStarter, @ASU_SFIS, and @AAPSciences 💙
ALT Promotional graphic for One Million Acts of Science Citizen Science Month in April, featuring logos of SciStarter, NORD, and Living Rare Study.
Rare Diseases retweeted
What is the most common symptom of invasive ductal carcinoma? Is it curable? And how can you reduce your risk of developing it?
Our expert answers these questions and more: brnw.ch/21wRZpl #EndCancer
Rare Diseases retweeted
CRU exec director @ZivGanOr was in Vienna for #RARE2025 to discuss his lab’s work understanding #GBA1, the genetic mutation behind a rare subtype of #Parkinsons. The CRU works to offer the most advanced investigational therapies for rare diseases, including RNA and gene therapies
Rare Diseases retweeted
Coping with adversity when living with a rare disease bit.ly/3PUz6c8. Please read now at PatientWorthy.com.