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Topic :

Syndromes

NASDAQ, TCRX

#Event #Phase 1 #Syndromes #Trending News

TScan Therapeutics Presents Preliminary Phase 1 Clinical Results on TSC-100 and TSC-101 at the American Society of Gene & Cell Therapy 26th Annual Meeting

May 17, 2023
07:00

EST

Poster highlights Phase 1 results following treatment with TSC-100 and TSC-101 after hematopoietic cell transplantation Company to host a virtual KOL event featuring Monzr M. Al Malki, M.D., to discuss highlights from the meeting and preliminary data today, Wednesday, May 17th at 5:30 p.m. ET WALTHAM, Mass., May 17,

No Comments

NASDAQ, RGNX

#Syndromes #Trending News

REGENXBIO Announces NAV® Technology Platform Will Support Bespoke Gene Therapy Consortium’s First Rare Disease Clinical Portfolio

May 16, 2023
17:52

EST

Research in MPS IVA led by REGENXBIO scientists moving into clinic Donation of NAV® Technology licenses will support development of gene therapies for rare diseases ROCKVILLE, Md., May 16, 2023 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) today announced that preclinical research in Mucopolysaccharidosis type IVA (MPS IVA), also known

No Comments

NASDAQ, XFOR

#Phase 3 #Positive Clinical Trial Results #Syndromes #Trending News #Upcoming Presentation

X4 Pharmaceuticals Announces Positive Phase 3 Results Showing Mavorixafor Reduced the Rate, Severity, and Duration of Infections vs. Placebo in Participants Diagnosed with WHIM Syndrome

May 16, 2023
16:01

EST

~60% reduction in annualized infection rate seen in the mavorixafor arm vs. placebo (p<0.01) >75% reduction in the percentage of individuals experiencing severe infections (Grade 3 or higher) in the mavorixafor group vs. placebo group >70% reduction in mean total days with infections (2 weeks on mavorixafor treatment vs.

No Comments

NASDAQ, XFOR

#Private Placement at Market Price #Syndromes #Trending News

X4 Pharmaceuticals Announces $65 Million Private Placement Priced At-the-Market

May 16, 2023
08:30

EST

BOSTON, May 16, 2023 (GLOBE NEWSWIRE) — X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small molecule therapeutics to benefit people with rare diseases of the immune system, today announced that it has agreed to sell an aggregate of 42,784,203 shares of common

No Comments

CYTO, NASDAQ

#Financial Data #Letter to Shareholders #Syndromes #Trending News

Altamira Therapeutics Provides Business Update, Reports FY 2022 Financial Results

May 16, 2023
07:30

EST

Management will host an investor conference call today, May 16th, at 8 a.m. Eastern Company is advancing OligoPhore™ / SemaPhore™ platform for extrahepatic RNA delivery and efficient endosomal release; expects first research collaborations with biopharmaceutical companies in 2023 Filed IND submission with FDA for AM-125 in acute vestibular syndrome

No Comments

NASDAQ, NMTR

#Financial Data #Letter to Shareholders #Syndromes #Trending News

9 Meters Biopharma Provides Business Update and Reports Financial Results for First Quarter 2023

May 15, 2023
17:15

EST

RALEIGH, NC / ACCESSWIRE / May 15, 2023 / 9 Meters Biopharma, Inc. (Nasdaq:NMTR), a clinical-stage company pioneering novel treatments for people with rare or debilitating digestive diseases, today provided a business update and announced financial results for the first quarter ended March 31, 2023. John Temperato, President &

No Comments

ELOX, NASDAQ

#Featured #Financial Data #Letter to Shareholders #Syndromes #Trending News

Eloxx Pharmaceuticals Reports First Quarter 2023 Financial and Operating Results and Provides Business Update

May 15, 2023
16:30

EST

Topline results to include biopsy results for the Phase 2 clinical study evaluating ELX-02 for the potential treatment of Alport syndrome expected in first half of 2023; meaningful protein reduction has been observed in one patient to date Received U.S. Food and Drug Administration (FDA) clearance to begin a

No Comments

NASDAQ, ZYNE

#Cannabis #Financial Data #Syndromes #Trending News

Zynerba Pharmaceuticals Reports First Quarter 2023 Financial Results and Operational Highlights

May 15, 2023
07:00

EST

Enrollment continues in RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel™ in patients with Fragile X syndrome; topline results expected first-half 2024 $44.4 million in cash and cash equivalents at March 31, 2023; Cash runway to mid-year 2024 Proposal for a reverse stock split is on the agenda

No Comments

JSPR, NASDAQ

#Financial Data #Letter to Shareholders #Syndromes #Trending News

Jasper Therapeutics Reports First Quarter 2023 Financial Results and Provides a Business Update

May 12, 2023
16:05

EST

Announced Planned Development Expansion of Briquilimab in Chronic Spontaneous Urticaria Presented Positive Follow-up Clinical Data from Investigator-Sponsored Study of Briquilimab Conditioning in Fanconi Anemia Patients at the 2023 Transplantation & Cellular Therapy Meetings of the ASTCT and CIBMTR Announced Positive Clinical Data from a Phase I/II Trial of Briquilimab

No Comments

NASDAQ, SLGL

#Corporate Presentation #Financial Data #Syndromes

Sol-Gel Technologies Reports First Quarter 2023 Financial Results and Provides Corporate Update

May 12, 2023
07:00

EST

Positive trends continue for TWYNEO® and EPSOLAY® with recurrent prescriber bases at 98% for TWYNEO and 92% for EPSOLAY during Q1 2023 Sol-Gel on track to advance Orphan Drug candidate, SGT-610 (patidegib) for Gorlin syndrome into Phase 3 testing in late 2023 Phase 1 trial for SGT-210 (erlotinib) demonstrated

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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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