Rare Diseases

Pharvaris Reports Positive Phase 2 Results for Oral Hereditary Angioedema (HAE) Drug Deucrictibant in The Lancet Haematology - For More Information Visit shorturl.at/7ByNY @pharvaris #Hereditary_Angioedema #HAE #Rare_Diseases #Orphan_Drugs @TheLancetHaem

IntraBio Announces Submission of Supplemental New Drug Application for Levacetylleucine for Ataxia-Telangiectasia - For More Information Visit shorturl.at/DeqHp @IntraBio #Ataxia_Telangiectasia #A_T

Dartmouth Study Finds Kidney Transplants May Extend Survival by Nearly 20 Years in Cystic Fibrosis Patients - For More Information Visit shorturl.at/Mn1A9 @GeiselMed #Cystic_Fibrosis #Rare_Diseases #Orphan_Drugs

Review Finds VPRIV Safe and Effective for Children and Adolescents with Gaucher Disease Type 1 with potential benefits in type 3 - For More Information Visit shorturl.at/CG5Wq @TakedaPharma #Gaucher_Disease #GD1 #Rare_Diseases #Orphan_Drugs #VPRIV #Takeda

Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity - For More Information Visit shorturl.at/ZzNDK @RhythmPharma #Rare_Diseases #FDA #Hypothalamic_Obesity #HyOb #Orphan_Drugs #IMCIVREE

AL-S Pharma’s AP-101 Shows Promising Disease-Modifying Effects and Survival Benefit in Phase 2 ALS Trial - For More Information Visit shorturl.at/DLHqe #AL_S_Pharma #Amyotrophic_Lateral_Sclerosis #ALS

Remix Therapeutics Granted FDA Fast Track Designation for REM-422 in the Treatment of Recurrent, Metastatic or Unresectable Adenoid Cystic Carcinoma - For More Information Visit shorturl.at/h4Mgg #FDA @RemixThera #Rare_Diseases #Adenoid_Cystic_Carcinoma

Shionogi Announces First Patients Enrolled in Global Phase 2 Clinical Trial in Adults with Late-Onset Pompe Disease - For More Information Visit shorturl.at/kAKbF @ShionogiUS #Pompe_Disease #LOPD #Rare_Diseases

Sarepta Seeks Full FDA Approval for Duchenne Therapies AMONDYS 45 and VYONDYS 53 Using ESSENCE and Real-World Data - For More Information Visit shorturl.at/tpqjh @Sarepta #Rare_Diseases #DMD #FDA #Duchenne #Orphan_Drugs #Muscular_Dystrophy

Grace Therapeutics to Present GTx-104 Data Addressing aSAH Treatment Gaps at Major March 2026 Medical Conferences - For More Information Visit shorturl.at/4D2U5 #Grace_Therapeutics #Rare_Diseases #aSAH

Lynavoy (Linerixibat) Approved by the US FDA for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (PBC) - For More Information Visit shorturl.at/5Awgw @GSK #Primary_Biliary_Cholangitis #PBC #FDA #Rare_Diseases #Orphan_Drugs #Glaxo_Smith_Kline

Aro Biotherapeutics Reports Positive Phase 1b Results for ABX1100 in Late-Onset Pompe Disease, Showing Strong Lung Function Improvement - For More Information Visit shorturl.at/fziLT @AroBioTx #Pompe_Disease #LOPD #Rare_Diseases #Orphan_Drugs #neuromuscular_disorder

Sanofi’s Venglustat Earns Breakthrough Therapy Designation in the US For Gaucher Disease Type 3 - For More Information Visit shorturl.at/tYR50 @sanofi #Gaucher_Disease #Rare_Diseases #Orphan_Drugs #GD3

FDA Feedback Supports Extension Phase for Jaguar Health’s Clinical Trial of Crofelemer in the Treatment of Microvillus Inclusion Disease (MVID) - For More Information Visit shorturl.at/k544N @Jaguar_Health #MVID #Rare_Diseases #Microvillus_Inclusion_Disease #FDA

Candel’s Aglatimagene Therapy Shows Extended Survival Benefits in Advanced NSCLC Patients, Paving the Way for Phase 3 Trial - For More Information Visit shorturl.at/kvV52 @CandelTx #Lung_Cancer #NSCLC #Rare_Diseases #Orphan_Drugs #Cancer_research #Candel

Polaryx Therapeutics Receives FDA Fast Track Designation for PLX‑200 in Late‑Infantile Neuronal Ceroid Lipofuscinosis (CLN2) - For More Information Visit shorturl.at/GpZHY @PolaryxTx #Rare_Diseases #CLN2 #FDA #Neuronal_Ceroid_Lipofuscinosis #Orphan_Drugs

Ascendis Pharma Reports Positive Week 52 Phase 2 Results Showing TransCon hGH Matches Daily Somatropin in Children with Turner Syndrome - For More Information Visit shorturl.at/oQgx6 @AscendisPharma #Rare_diseases #Orphan_Drugs #Turner_Syndrome #Pediatric_diseases

CytomX Reports Positive Phase 1 Data for Varseta-M in Late-Line Metastatic Colorectal Cancer, Plans FDA Discussions for Registrational Path - For More Information Visit shorturl.at/d0M15 @CytomXInc #mCRC #FDA #Colorectal_Cancer #Rare_Diseases #Orphan_Drugs

Sarepta Begins ENDEAVOR Cohort 8 to Test Sirolimus Protocol with ELEVIDYS in Non-Ambulatory Duchenne Patients - For More Information Visit shorturl.at/7J8X3 @Sarepta #Duchenne #Rare_Diseases #DMD #Muscular_Dystrophy #Orphan_Drugs #ELEVIDYS #Sarepta

@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