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Stay informed: Whether you’re a clinician, patient, carer, or patient representative, our ERN-RND #newsletter is your go-to source for the latest updates on Rare Neurological Diseases in Europe: www.ern-rnd.eu?mailpoet_rou...

#RareDisease #RareNeurologicalDisease #Neurology #RareNeurology
November 6, 2025 at 3:27 PM
healthdivine.bsky.social
🧠 Targeting rare movement disorders! Novel therapies for choreoathetosis are reshaping neurology care.
#RareNeurology #PatientCare #PharmaResearch

www.marketresearchfuture.com/reports/paro...
Paroxysmal Choreoathetosis Market Size, Growth Outlook 2035
Paroxysmal Choreoathetosis Market growth is projected to reach USD 450.0 Billion, at a 7.3% CAGR by driving industry size, share, top company analysis, segments research, trends and forecast report 20...
www.marketresearchfuture.com
September 15, 2025 at 7:44 AM
checkrare.bsky.social
Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).

checkrare.com/a-familys-ex...

#CheckRare #ALSP #RareNeurology #RareGenetic #Disease
June 12, 2025 at 2:37 PM
checkrare.bsky.social
💡Rare Disease Spotlight: SYNGAP1-Related Disorders

Learn more about this rare disease with our latest article checkrare.com/the-current-...

#CheckRare #RareDisease #SYNGAP1 #RareNeurology #RareGenetic
June 11, 2025 at 3:53 PM
checkrare.bsky.social
Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, discusses the company’s current drug development programs.

checkrare.com/camp4-therap...

#CheckRare #RareNeurology #RareMetabolic #UCD #SYNGAP1
May 29, 2025 at 7:09 PM
checkrare.bsky.social
Norman Putzki, MD, Global Development Head of Neuroscience and Gene Therapy at Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for spinal muscular atrophy (SMA).

checkrare.com/positive-saf...

#CheckRare #SMA #RareNeurology #RareMusculoskeletal
May 19, 2025 at 2:26 PM
checkrare.bsky.social
Sonia Gobeil, co-founder of Ataxia of Charlevoix-Saguenay Foundation, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS).

checkrare.com/ongoing-rese...

#CheckRare #RareNeurology #RareMusculoskeletal #ARSACS
May 16, 2025 at 3:30 AM
checkrare.bsky.social
The U.S. FDA has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis.

checkrare.com/fda-approves...

#CheckRare #FDAApproval #MyastheniaGravis #RareNeurology #RareMusculoskeletal
FDA Approves Myasthenia Gravis Treatment Nipocalimab
The U.S. Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG).
checkrare.com
May 6, 2025 at 11:09 PM
checkrare.bsky.social
Sindhu Ramchandren, MD, Johnson & Johnson, discusses updated results from the Vivacity-MG3 clinical trial in patients with generalized myasthenia gravis (MG).

checkrare.com/efficacy-of-...

#CheckRare #RareNeurology #MyastheniaGravis
April 25, 2025 at 11:41 PM
checkrare.bsky.social
Aravindhan Veerapandiyan, MD, discusses results from the phase 1/2 EXPLORE44 clinical trial for Duchenne muscular dystrophy (DMD).

checkrare.com/results-from...

#CheckRare #RareMusculoskeletal #RareNeurology #RareGenetic #DMD #Duchenne #RareDisease
April 2, 2025 at 6:54 PM
checkrare.bsky.social
💡Rare Disease Spotlight: ALS

Learn more about this rare disease with our latest article checkrare.com/diseases/can...

#CheckRare #RareDisease #ALS #RareMusculoskeletal #RareNeurology
Cancers
There are over 200 different types of rare cancers, but only a small number of people are diagnosed with each type.
checkrare.com
March 26, 2025 at 5:44 PM
checkrare.bsky.social
Alan Percy, MD, Pediatric Neurologist at the University of Alabama at Birmingham, discusses results from the DAFFODIL study evaluating long-term safety of trofinetide in girls ages two to four years with Rett syndrome.

checkrare.com/long-term-sa...

#CheckRare #RettSyndrome #RareNeurology
March 19, 2025 at 3:05 AM
checkrare.bsky.social
💡Rare Disease Spotlight: Sanfilippo Syndrome Type A

Learn more about this rare disease with our latest article checkrare.com/gene-therapy...

#CheckRare #RareDisease #MPSIIIA #RareNeurology
March 4, 2025 at 11:17 PM
checkrare.bsky.social
Carlo Antozzi, MD, discusses results from the phase 3 Vivacity-MG3 study of nipocalimab in antibody positive adults with generalized myasthenia gravis (MG).

checkrare.com/results-from...

#CheckRare #MyastheniaGravis #RareNeurology #RareMusculoskeletal
Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis
Carlo Antozzi, MD, discusses results from the phase 3 Vivacity-MG3 study of nipocalimab in antibody positive adults with MG.
checkrare.com
March 3, 2025 at 8:21 PM
efna.net
Missed last week's meeting of the MEP Interest Group on Brain Health & Neurological Conditions? No problem, you can watch a reply of the event on our YouTube Channel now: youtu.be/1Zr0zm8ILO0?...
#RareDiseaseDay #RareNeurology
Shaping the Future: Policy Responses to Rare Neurological Challenges
YouTube video by European Federation of Neurological Associations
youtu.be
February 27, 2025 at 4:56 PM
efna.net
Tomorrow is #RareDiseaseDay & we'll be marking the day by sharing some facts about the impact of rare neurological conditions. You can get involved by sharing your story with the #rarediseaseday and #rareneurology hashtags. You'll find lots of resources at www.rarediseaseday.org/downloads/
February 27, 2025 at 3:50 PM
checkrare.bsky.social
Michael Panzara, MD, discusses topline results of the Honeycomb trial for GRIN-related neurodevelopmental disorder with GoF variants.

checkrare.com/topline-resu...

#GRINDisorders #RareNeurology #ClinicalTrials
Topline Results of the Honeycomb Trial for GRIN-related Neurodevelopmental Disorders
Michael Panzara, MD, discusses topline results of the Honeycomb trial for GRIN-related neurodevelopmental disorder with Go) variants.
checkrare.com
February 19, 2025 at 9:03 PM
efna.net
Join us on 19 February for a meeting of the MEP Interest Group on Brain Health & Neurological Conditions, 'Shaping the Future: Policy Responses to Rare Neurological Challenges' at the EU Parliament and online.
Register now: www.efna.net/mep-february...

#neurology #RareNeurology #RareDiseaseDay
January 30, 2025 at 2:53 PM