Real-world and clinical trial data underscore long-term safety and effectiveness in pediatric and adult NPC patientsCELEBRATION, Fla., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the presentation of four posters highlighting positive new data on MIPLYFFA® (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at th

Brepocitinib 45 mg significantly improved cutaneous sarcoidosis disease activity, achieving a 22.3-point improvement in mean CSAMI-A at Week 16 versus a 0.7-point improvement in placebo (Δ 21.6 P<0.0001)All brepocitinib 45 mg patients achieved a clinically meaningful response, with 100% demonstrating at least a 10-point improvement on CSAMI-AOn the Investigator’s Global Assessment (IGA), 69% percent of brepocitinib 45 mg patients compared to 0% of placebo patients achieved the gold standard two-

PHocus enrollment completed in under 12 months from first patient dosed, representing a rapid enrollment pace in pulmonary hypertension associated with interstitial lung diseaseTopline results on track for second half of calendar year 2026 WALTHAM, Mass., Feb. 06, 2026 (GLOBE NEWSWIRE) -- Pulmovant, a clinical-stage biotechnology company committed to transforming the lives of patients with pulmonary diseases, and a Roivant (Nasdaq: ROIV) company, today announced that it has completed enrollme

~ Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3 levels maintained post-dosing, regardless of enzyme replacement therapy status across all cohorts ~ ~ Six of 11 patients have discontinued enzyme replacement therapy as of data cutoff date ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 06, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative ther

DPTX3186, a first-in-class beta-catenin condensate modulator, received Orphan Drug Designation and Fast Track Designation from FDAClinical entry validates condensate modulator discovery platform and its potential to address historically intractable targets through a novel mechanism BOSTON, Feb. 04, 2026 (GLOBE NEWSWIRE) -- Dewpoint Therapeutics, Inc., a clinical-stage biotechnology company pioneering condensate-modulating therapeutics, today announced that the first patient has been dosed in

Feb. 4, 2026 -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its novel oral therapy targeting a fatal form of heart disease in Duchenne muscular dystrophy (DMD) patients. The FDA's Fast Track program facilitates the development and expedites the review of a drug designed to treat a serious or life-thre

February 4, 2026 Biodexa Announces Exclusive License of Otsuka’s OPB-171775, a potent Phase 1 ready Molecular Glue for GIST Novel Mechanism of Action Shown to be Effective in TKI Resistant PDX Models Cardiff, UK – February 4, 2026 – Biodexa Pharmaceuticals PLC (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of rare diseases with unmet medical needs, today announced the closing of an exclusive license with Otsuka Pharmac

Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AFTX-201, an investigational genetic medicine for the treatment of BAG3 DCM. “We are grateful to the FDA for the timely review of our IND application f

Tr1X, a clinical-stage autoimmune and inflammatory disease company developing Type 1 regulatory T (Tr1) cell therapies, today announced the first clinical data from its ongoing Phase 1 study of TRX103 in patients with hematologic malignancies undergoing HLA-mismatched hematopoietic peripheral stem cell transplantation (HSCT), presented at the 2026 ASTCT/CIBMTR Tandem Meetings. The data represents the first clinical results for TRX103, an investigational, off-the-shelf engineered Tr1 cell therap

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has granted approval for its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT. Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA (siRNA) therapeu