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Syndromes

NASDAQ, QNRX

#New Clinical Trial Data #Syndromes #Trending News

Quoin Pharmaceuticals Announces 50% Enrollment in Open Label Netherton Syndrome Clinical Trial

May 24, 2023
08:15

EST

ASHBURN, Va., May 24, 2023 (GLOBE NEWSWIRE) — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a clinical stage, specialty pharmaceutical company focused on rare and orphan diseases, announces that its open label study evaluating QRX003 as a treatment for Netherton Syndrome has reached 50% enrollment. The company’s

No Comments

OTC, TRYPF

#FDA Application Submitted #Phase 2 #Syndromes

Tryp Therapeutics Submits Investigational New Drug (IND) Application to US FDA for Planned Phase 2a Clinical Trial in Patients With IBS at Massachusetts General Hospital

May 24, 2023
06:00

EST

KELOWNA, BC / ACCESSWIRE / May 24, 2023 / Tryp Therapeutics, Inc. (CSE:TRYP)(OTCQB:TRYPF) (Tryp or the Company), a clinical-stage biotechnology company focused on developing intravenous-infused psilocin (the active metabolite of psilocybin) for diseases with high unmet medical needs, today announced it has submitted an Investigational New Drug (IND) application

No Comments

OTC, TRYPF

#FDA Application Submitted #Phase 2 #Syndromes

Tryp Therapeutics Submits Investigational New Drug (IND) Application to US FDA for Planned Phase 2a Clinical Trial in Patients With IBS at Massachusetts General Hospital

May 24, 2023
06:00

EST

KELOWNA, BC / ACCESSWIRE / May 24, 2023 / Tryp Therapeutics, Inc. (CSE:TRYP)(OTCQB:TRYPF) (“Tryp” or the “Company”), a clinical-stage biotechnology company focused on developing intravenous-infused psilocin (the active metabolite of psilocybin) for diseases with high unmet medical needs, today announced it has submitted an Investigational New Drug (IND) application

No Comments

NASDAQ, RGNX

#FDA #Syndromes

REGENXBIO Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RGX-121 Gene Therapy for Hunter Syndrome

May 23, 2023
07:05

EST

RMAT recognizes that the preliminary clinical evidence from RGX-121, a potential one-time AAV Therapeutic, indicates the potential to address unmet medical needs for MPS II RMAT designation is for gene therapies intended to treat or cure serious condition in order to expedite the drug development and review processes CAMPSIITE™ trial

No Comments

NASDAQ, ZYNE

#Cannabis #Event #New Clinical Trial Data #Syndromes

Zynerba Pharmaceuticals Announces Podium and Poster Presentations at the American Society of Clinical Psychopharmacology Annual Meeting

May 23, 2023
07:00

EST

DEVON, Pa., May 23, 2023 (GLOBE NEWSWIRE) — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today announced an oral podium presentation and poster presentations at the American Society of Clinical Psychopharmacology Annual Meeting (ASCP 2023), being held May 30

No Comments

NASDAQ, RDHL

#Letter to Shareholders #Syndromes

RedHill Provides R&D Update

May 22, 2023
07:00

EST

RedHill has elected to terminate RHB-204’s U.S. Phase 3 study for Non-tuberculosis Mycobacteria (NTM) disease due to low accrual rate and plans to shift resources to more rapidly advance RHB-107 late-stage development for outpatient treatment of COVID-19 RedHill’s advanced pipeline also includes: Opaganib – in collaboration with the U.S.

No Comments

NASDAQ, TSHA

#Event #New Clinical Trial Data #Syndromes #Trending News

Taysha Gene Therapies Presents Preclinical Data on TSHA-102 for Rett Syndrome Demonstrating Cellular Regulation of MeCP2 Expression in Key Mouse Models at the American Society of Gene and Cell Therapy 26th Annual Meeting

May 19, 2023
08:00

EST

New preclinical data after neonatal administration in wild-type mice showed no detectable impact on survival, neurobehavioral functions and overall health, suggesting TSHA-102, engineered with novel miRARE technology, avoided toxic overexpression of MeCP2 within cells already expressing MeCP2 Data reinforce previous findings in Mecp2–/Y knockout mice demonstrating TSHA-102 regulated cellular

No Comments

ATHX, NASDAQ

#Financial Data #Syndromes #Trending News

Athersys Reports First Quarter 2023 Financial Results and Business Highlights

May 18, 2023
09:58

EST

CLEVELAND–(BUSINESS WIRE)–Athersys, Inc. (NASDAQ: ATHX), a regenerative medicine company developing MultiStem® (invimestrocel) cell therapy for critical care indications, announced on Monday, May 15th financial results for the three months ended March 31, 2023 and provided a business update. First Quarter 2023 and Recent Corporate and Operational Highlights Continued reducing

No Comments

BMRA, NASDAQ

#Syndromes #Trending News

inFoods® IBS Test Now Validated for Use with Simple Finger Stick Whole Blood Collection Technology

May 18, 2023
08:19

EST

Gastro Health, a leading gastroenterology group, plans to utilize this simplified blood collection method when recommending the inFoods® IBS to their patients Biomerica also announced that inFoods® IBS clinical trial data was presented at the DDW conference May 8th.The data showed that the inFoods® IBS treatment diet significantly improved

No Comments

NASDAQ, RARE

#Expansion #FDA #Phase 1/2 #Syndromes #Trending News

Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S.

May 17, 2023
08:00

EST

NOVATO, Calif., May 17, 2023 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) has reviewed and agreed to a protocol amendment to the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome that enables the company to

No Comments

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Trending News

Iovance Biotherapeutics Announces U.S. Food and Drug Administration Acceptance of the Biologics License Application of Lifileucel for the Treatment of Advanced Melanoma

05/26/2023

SILVERCORP DECLARES SEMI-ANNUAL DIVIDEND OF US$0.0125 PER SHARE

05/26/2023

Halo Collective Provides Update on Status of Financial Statements

05/26/2023

Molecular Templates to Present on Phase I Dose Escalation Study of MT-6402 at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting

05/26/2023

US Investors May Now Have Trading Access for Genius Group Shares on Upstream through US Broker Dealer

05/26/2023

Most read news

New England Journal of Medicine Publishes Results from Phase 3 Induction and Maintenance Programs Evaluating Upadacitinib (RINVOQ®) in Crohn’s Disease

05/25/2023

Shareholder Alert: Ademi LLP investigates whether CohBar, Inc. has obtained a Fair Price in its transaction with Morphogenesis, Inc.

05/24/2023

Avante Announces Private Placement Closing

05/24/2023

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